Camargo 505(b)(2) Blog

The Race to Get a Cannabidiol Product Approved: Why Is It Taking So Long?

Several companies have been competing to get the first cannabidiol product to market. Both investor and patient interest are high but the recent news includes more setbacks. What is different about cannabidiol (CBD) products and what could Zynerba Pharmaceuticals, Inc. have done differently? The Cannabidiol Pipeline As we have previously blogged, there are currently no […]
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Navigating Clinical Holds

Sponsors spend countless hours developing Investigational New Drug (IND) applications, which are the US FDA’s regulatory gateways for conducting clinical trials of investigational drug and drug-device combination products. The stakes are high for companies as they submit their initial IND, as the ability to start clinical trials hinges on the activation of the IND. But […]
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De-risking Drug Development

High-level Reasoning and Technical Methodology for Evaluating a Program’s Risk From a distance, 505(b)(2) product development can seem very straightforward for products that have been on the market or in clinical use for long periods of time. However, these types of products can often present the greatest challenges when trying to modify or improve performance. […]
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Stability Requirements in the 505(b)(2) Space: Why, What, When, How

Stability Requirements in the 505(b)(2) Space: Why, What, When, How Hurry up and wait. That’s the seemingly eternal impact of developmental stability on the new drug development process. The question always asked is: “How can the developer minimize the wait part?” At the top level you can’t. It will always take six months to get […]
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Deuterization: Is it Enough to Get 5- or 7-Year Exclusivity for a 505(b)(2) Product?

Deuterization: Is it Enough to Get 5- or 7-Year Exclusivity for a 505(b)(2) Product? As the 505(b)(2) experts, Camargo has received several enquiries about developing deuterated drugs as a means of achieving sustained-release properties for a product. The approval of Austedo (deutetrabenazine; Teva Pharmaceuticals, Inc.; NDA 208082), marked the first FDA approval of a deuterated […]
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What Went Wrong? Make Sure Your Bridge Stays Intact, CMC and Dissolution

Make Sure Your Bridge Stays Intact: CMC and Dissolution in 505(b)(2) Development One of the most common reasons for a company developing a drug to receive a Refuse to File letter from the FDA is for problems with Chemistry, Manufacturing, and Controls (CMC). Camargo has a robust in-house CMC team to help address and prevent […]
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Camargo Counsel: The New Reality of Generics under GDUFA

How to Navigate the Impact of GDUFA From recent news, we know that the FDA has been under increasing pressure to reduce the timing of its reviews while maintaining the highest possible standards for safety and efficacy of the products it approves. This is the reason for the PDUFA and now the GDUFA goals. FDANews.com […]
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PREA Requirements for Biosimilar and Interchangeable Biological Products

In general, under the Pediatric Research Equity Act (PREA), submission of an initial pediatric study plan (iPSP) is required for a drug or biological product that includes a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration. Submission of an iPSP is required 60 calendar days after the […]
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Brexit and EMA: The Changes Have Begun

After months of speculation, the first round of changes for pharmaceutical manufacturers arising from the planned withdrawal of the United Kingdom of Great Britain and Northern Ireland (UK) from the European Union (EU) have arrived. And the changes are major for companies based in the UK that currently market or plan to market centrally authorised […]
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What Went Wrong? Important Outcomes of a Successful Pre-IND Meeting

We are pleased to present the second episode in a new format and series we’re adding to our 505(b)(2) Blog: a video blog / podcast called What Went Wrong? For our second episode, Ken Phelps, CEO, and Dr. Ruth Stevens, CSO, Camargo Pharmaceutical Services, discuss important outcomes for a successful Pre-IND meeting, which is essential […]
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Due Diligence Assessment: Determining a Drug Product’s Potential Value

Due Diligence Assessment: Determining a Drug Product’s Value The risk involved with an asset needs to be assessed before a sale. Whether from the buy or from the sell side, it pays to understand how much an asset is worth. For those involved with in-licensing or out-licensing, a properly performed Due Diligence Assessment places a […]
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The Value of a Strategic Assessment: Aligning for Success from the Start

The Value of a Strategic Assessment The first step for every wise drug developer beginning a drug development program is to determine the feasibility of a proposed product by asking several high-level questions: Is the product scientifically, medically, and commercially viable? What are the regulatory requirements and potential hurdles? What nonclinical and/or clinical studies will […]
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Abuse-Deterrent Opioids – The Insider’s Guide to Innovation and Exclusivity in a Changing Regulatory Landscape

Abuse-Deterrent Opioids – The Insider’s Guide to Innovation and Exclusivity in a Changing Regulatory Landscape As discussed in a previous blog post (here), the 505(b)(2) development pathway provides a flexible path for developers of new abuse-deterrent formulations. The FDA has shown significant motivation and flexibility in its drive to work with innovators to address the […]
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Special Protocol Assessment: Is It Important for Your Drug Development Program?

The overarching goal of the Special Protocol Assessment Draft Guidance for Industry May 2016 (HHS, FDA, CDER, & CBER) is to improve the quality of new drug applications (NDAs) and biologic license applications (BLAs) by providing more certainty in the clinical protocol design process. Among its several purposes, the Special Protocol Assessment (SPA) affords an […]
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Getting Cannabis-related Products Approved: the 505(b)(2) 4-20 Projects

Several states in the US have already passed laws that remove state restrictions on the medical use of marijuana and its derivatives, and more states are considering such action. The market for medical marijuana in the US is expected to surpass $20 billion by 2020. However, federal restrictions prevent the use of medical marijuana as […]
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Shortening the Review Clock: the Latest on Priority Review Vouchers

The Latest on Priority Review Vouchers Priority review vouchers (PRVs) are a valuable incentive available to companies upon approval of novel drugs to treat rare pediatric diseases or certain tropical diseases. These vouchers can be used on a subsequent application to speed the review process and are an asset that can be sold or transferred […]
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DESI Drugs: Potential Targets for Quick Approvals

DESI Drugs: potential targets for quick approvals Drugs that are on the market but are not approved by the FDA are more common than you might think. Even some physicians might be unaware that the drug they are prescribing has not been approved, meaning it has not undergone the rigorous standards of safety and efficacy […]
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Fixed-Dose Combination Products – Navigating the Combination Rule

Fixed-dose combination products (FDCs), or drugs containing multiple active ingredients, offer benefits to pharmaceutical companies and patients. For Pharma, creative matching of multiple APIs can open new markets, while for patients, FDCs can offer convenience and therapeutic benefits. Often, FDCs are composed of previously approved agents, and the 505(b)(2) pathway is commonly used for these […]
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505(b)(2) Approval Times: The Real Scoop

The Approval Time for 505(b)(2) and 505(b)(1) NME Products Is Similar A recent article by the Tufts Center for the Study of Drug Development (summarized here) reported that approval times for New Molecular Entities (NMEs) approved via the 505(b)(2) pathway are nearly 5 months longer than that of NMEs approved by other pathways, 505(b)(1), or […]
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Pre-IND Meetings: How to Achieve Success for 505(b)(2)

One of the greatest mistakes that the Sponsor of a 505(b)(2) can make is to have an unsuccessful Pre-IND meeting. Common errors occur at the Pre-IND meeting because Sponsors and CROs that are more familiar with traditional 505(b)(1) drug development programs fail to appreciate the different goals and the impact of a Pre-IND meeting on […]
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Camargo Counsel: The Cost of Wrong

Here at the Camargo 505(b)(2) blog, we are adding a new voice to mix in with the technical writing. These candid takes will be given by our experts in drug development and sprinkled in with the usual fare. Camargo Counsel is a venue for straightforward talk and advice related to drug development and 505(b)(2). With […]
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Demystifying Orange Book Designations: The New Referencing Approved Drug Products in ANDA Submissions Draft Guidance

Demystifying Orange Book Designations Since its first appearance in 1980, the Approved Drug Products With Therapeutic Equivalence Evaluations publication (commonly referred to as the Orange Book) has served as a gateway for the emergence of generic drug products via the 505(j) drug approval pathway (and some new drug products submitted through the 505(b)(2) approval pathway). […]
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Referencing a Listed Drug for the 505(b)(2) Pathway

Section 505(b)(2) of the Food, Drug, and Cosmetic Act describes a 505(b)(2) new drug application (NDA) as an application where at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference. This type of information […]
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Clinical Trials With Multiple Endpoints: Pitfalls and Management

Randomized controlled Phase 3 (and some Phase 2) studies are clinical trials that are designed to answer specific questions, such as whether the proposed drug is effective in treatment or prevention of a particular disease. As such, the primary endpoint is the most important one that will demonstrate the primary effects being sought by the […]
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Real-World Evidence: Can it Really Be Used For Drug Approvals?

With the signing of the 21st Century Cures Act, the US Congress tasked the FDA with developing a framework to evaluate how the use of data from sources other than traditional clinical trials may be used to support drug approvals. This framework will apply to post-market commitments and to new indications for approved drugs. This […]
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Electronic Submissions Update: End of FDA Paper Submissions Looms and What It Means

Electronic Submissions Update: the end of paper submissions looms closer, and requirements for Standardized Study Data go into effect: What that means for industry Under section 745A(a) of the FD&C Act, no earlier than 24 months after FDA issued the final guidance on December 2014, “Providing Regulatory Submissions in Electronic Format – Submissions Under Section […]
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Unforced Errors: FDA Refusal to File or Receive Letters

UNFORCED ERRORS: FDA Refusal to File or Receive Letters Few things can be more damaging to a pharmaceutical company than the refusal by the Food and Drug Administration (FDA) to review their New Drug Application (NDA) or Abbreviated New Drug Application (ANDA). When a company submits their application for authorization to market a new or […]
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On the Rise: 2016 505(b)(2) NDA Approvals

On the Rise: 2016 505(b)(2) NDA Approvals The total number of novel drug approvals in 2016 decreased by approximately half from last year (22 in 2016, from 45 in 2015) and is below the 10-year average of 29 drug approvals per year. Of these, how many were 505(b)(2)s? Were the number of 505(b)(2) approvals in […]
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Improvements and Updates to the FDA Inactive Ingredient Database

Improvements and Updates to the FDA Inactive Ingredient Database The FDA recently announced it has made corrections, updates, and additions of a backlog of formulations to the Inactive Ingredient Database (IID), an important modification for drug developers and development. The previous state of the IID had become an issue as excipient suppliers recommend the use […]
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Improving NDA Approval Odds for New Dosage Forms of Approved Products

Improving NDA Approval Odds for New Dosage Forms of Approved Products There are numerous reasons why a Sponsor may wish to market a new dosage form of an approved product. Aside from the obvious financial benefits to the sponsor, providing a more convenient and/or faster-acting dosage form of a well-chosen drug provides significant benefits for […]
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A New Indication for an Old Drug. What Could Go Wrong?

A New Indication for an Old Drug. What Could Go Wrong? Desmopressin (DDAVP®; Ferring Pharmaceuticals Inc) was approved in the US in 1978. DDAVP is currently approved to treat central diabetes insipidus, hemophilia A, type 1 von Willebrand’s disease, nocturnal enuresis (bedwetting) in children, and as a diagnostic test to measure renal concentrating abilities. Desmopressin […]
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Dramatically Decrease Drug Development Costs Through Literature-Only 505(b)(2) NDA Submissions

Dramatically Decrease Drug Development Costs Through Literature-Only 505(b)(2) NDA Submissions How would you like to get an NDA approved without conducting any clinical studies? Camargo presented a poster on literature-only 505(b)(2) New Drug Application (NDA) approvals at the recent 2016 AAPS annual meeting and exposition in Denver, Colorado. Individuals from the FDA and the pharmaceutical […]
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PRO-CTCAE: Improving Oncology Drug Development

Patient-reported outcomes (PROs) provide valuable tools for collecting information on subjective symptomatic effects during clinical trials. They are considered the gold standard for the assessment of health-related quality of life, treatment preferences, and satisfaction with care. PRO results from a well-defined and reliable PRO instrument can be used to support claims in product labeling, which […]
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Expedited Approval of FDA-approved Drugs in Australia

Expedited Approval of FDA-approved drugs in Australia: New Market Opportunities for Drugs and Devices In the past, after gaining approval for a drug/device in the United States, subsequent approval in Australia involved significant duplicated effort and additional regulatory hurdles. This has resulted in a lack of incentive for pharmaceutical companies and delays of up to […]
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505(b)(2) Application Changes: What You Need to Know

505(b)(2) Application Changes: What You Need to Know Title XI of the Medicare Prescription Drug, Improvement, and Modernization Act (MMA) of 2003 was enacted in order to address concerns that had potential to delay access to more affordable drugs. The FDA has been implementing the MMA via the statute since it was enacted in 2003. […]
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How Many FDA Premarket Applications Are Necessary For Your Combination Product?

This week, we are pleased to share an article written by Camargo Pharmaceutical Services’ new Medical Device expert, Dr. Girish Kumar, along with Dr. P.K. Narang, and published by Pharmaceutical Online, Med Device Online, and Bio Process Online. How Many FDA Premarket Applications Are Necessary For Your Combination Product? Often, an already-approved drug can further […]
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Extrapolation of Clinical Data for Pediatric Uses: Application for Medical Devices and Drug Products

Extrapolation of Clinical Data for Pediatric Uses: Application for Medical Devices and for Drug Products Extrapolation of Clinical Data for Medical Devices1 The Food and Drug Administration released the revised draft guidance entitled ‘‘Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices” on June 21, 2016 (Docket No. FDA–2015–D–1376). This guidance explains […]
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Importing Investigational Pharmaceuticals to the U.S.? Changes Are Coming

Importing Investigational Pharmaceuticals to the U.S.? Changes Are Coming The United States government is making ongoing, concerted efforts to improve the importation process for many products, including pharmaceuticals and medical devices. It’s important to stay on top of the new importation changes and their effect on business. Foreign biotech, device manufacturers, API producers, and pharmaceutical […]
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Risk Evaluation and Mitigation Strategies (REMS) Basics

The Food and Drug Administration is responsible for ensuring that human drugs are safe and effective, while also advancing public health by helping to speed product innovations. In determining if a drug should be marketed, the Agency must weigh the benefits of the therapy against its potential risks to the patient. If a drug or […]
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Effects on Exclusivity: The Biologics Price Competition and Innovation Act of 2009

On March 23, 2010, the U.S. FDA enacted the Biologics Price Competition and Innovation Act of 2009 (BPCIA) as part of the Patient Protection and Affordable Care Act (Public Law 111-148). The passing of BPCIA amended the definition of a “biological product” to include a “protein (except any chemically synthesized polypeptide)”, altering the way that […]
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Improving Drug Development ROI in 2017

Time to Pick the Low-Hanging Fruit: Improving Drug Development ROI in 2017 With forecasts of decreasing peak sales for late pipeline drugs, a logical way to increase the return on investment (ROI) for pharmaceutical companies is to develop products with lower research and development (R&D) costs. How can this be achieved in an environment with […]
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Leveraging Postmarketing Safety Data in 505(b)(2) Drug Development Programs

A significant part of the FDA’s charge is ensuring the safety of drugs available to the public. While a substantial part of the FDA’s efforts in guaranteeing public safety go into the safety assessment process during drug development, the process does not end with NDA approval. Postmarketing assessments such as pharmacovigilance programs play a critical […]
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Orphan Drug Development: Ensuring Best Time to Market

The Benefits of 505(b)(2) for Orphan Drug Development The Orphan Drug Designation Program, created by the Orphan Drug Act of 1983, provides significant financial incentives for the development of drugs for rare diseases. The potential incentives include tax credits for clinical trial costs, waiver of the Prescription Drug User Fee for NDA filing (~$2.4 million […]
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Pitfalls of Changing the Salt of a Listed Drug

Pitfalls of Changing the Salt of a Listed Drug The 505(b)(2) registration pathway for new drug products allows the applicant of the new drug product to reference the literature and the FDA’s findings of safety and/or effectiveness (e.g., as listed on the Listed Drug product’s label) to fulfill various registration requirements. From a nonclinical perspective, […]
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Abuse Deterrence Labeling – Generic vs 505(b)(2) Drug Development

Abuse Deterrence Labeling – Generic vs 505(b)(2) Drug Development With the ongoing opioid epidemic, drug abuse, diversion, and misuse are significant concerns for the FDA. The current opioid abuse problems also present significant opportunities for companies willing to explore new formulation technologies to deter abuse. Camargo has significant experience in opioid abuse and abuse-deterrent labeling. […]
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Protein Product 505(b)(2)s Face a Looming Application “Dead Zone”

The Biologics Price Competition and Innovation Act of 2009 (BPCIA) was enacted on March 23, 2010 as part of the Patient Protection and Affordable Care Act (Public Law 111-148). The BPCIA changed the regulation of certain protein products by amending the definition of a “biological product” to include a “protein (except any chemically synthesized polypeptide)”, […]
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The Prodrug Benefit of Utilizing the 505(b)(2) Pathway

Designing a new drug from scratch is costly and time-consuming. One attractive option to differentiate from currently marketed products is to chemically modify the characteristics of an existing drug to create a prodrug. Often a prodrug can improve the safety and efficacy of an approved therapeutic. Here, we discuss the basics of prodrugs, how the […]
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The New FDA Draft Guidance on Chewables

An idea for a more convenient dosage form for an existing drug product often presents an opportunity for a commercial advantage. Fortuitously, it also presents the possibility for using the 505(b)(2) regulatory pathway to product approval, which is often faster and less expensive than the 505(b)(1) route. Recently, FDA issued a brief draft guidance describing […]
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The GRAS Is Not Always Greener

The GRAS Is Not Always Greener: Why GRAS Status Does Not Guarantee Excipient Safety Many, if not most, 505(b)(2) submissions represent a change to an approved drug, usually involving a formulation change. Understandably, the focus of sponsors is often primarily on supporting the safety and efficacy of the active ingredient(s). However, the safety of the […]
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Back to Basics: 505(b)(2) FAQs Part 3: Regulatory Strategies

As the 505(b)(2) expert, Camargo is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Given the growing popularity of the 505(b)(2) pathway for approval of repurposed, reformulated, or unapproved-marketed products, we thought it would be worth providing a refresher. Here is Part […]
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Key Inflection Point in a Drug’s Time to Market: Choice of Regulatory Pathway

Traditional drug development follows a standard process beginning with nonclinical studies and moving into clinical studies, all with the purpose of proving a new drug candidate is safe and effective. When the U.S. opened up an alternate path, 505(b)(2), the primary purpose was to utilize existing data and knowledge, minimize costly animal and human studies, […]
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Back to Basics: 505(b)(2) FAQs Part 2: Clinical and Nonclinical Studies

As the 505(b)(2) expert, Camargo is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Given the growing popularity of the 505(b)(2) pathway for approval of repurposed, reformulated, or unapproved-marketed products, we thought it would be worth providing a refresher. Here is Part […]
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Drug Repositioning: Bringing New Life to Shelved Assets and Existing Drugs

Drug repositioning, also known as drug reprofiling or repurposing, has become an increasingly important part of the drug development process. This book examines the business, technical, scientific, and operational challenges and opportunities that drug repositioning offers. Readers will learn how to perform the latest experimental and computational methods that support drug repositioning, and detailed case […]
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Back to Basics: 505(b)(2) FAQs Part 1

As the 505(b)(2) expert, Camargo is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Given the growing popularity of the 505(b)(2) pathway for approval of repurposed, reformulated, or unapproved marketed products, we thought it would be worth providing a refresher. Here are […]
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The Regulation of Follow-On Biological Products via 505(b)(2)

Strike While the Iron is Hot In December 2015, the U.S. FDA granted approval for Eli Lilly and Company’s Basaglar (insulin glargine injection), a long-acting human insulin product indicated for glycemic control in patients with diabetes mellitus. Basaglar marked the first “follow-on” insulin therapy to be approved in the U.S. through a truncated review pathway […]
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Statistical Bootstrapping Method To Take the Uncertainty out of Drug Development

Statistical Bootstrapping Method Using a Bias-Corrected Acceleration Approach Well-reasoned and properly conducted statistical analyses can be essential to successful drug development, particularly in the context of manufacturing scale-up, process and component changes, and in comparisons with a reference product. Similarity tests between dissolution profiles steer the development path forward based on the quality and interpretation […]
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Fixed-Combination Drug Products: Are Phase 2 and 3 Studies Really Necessary?

Many fixed-dose drug-drug combination products arise from an observation that a synergistic effect occurs when two drugs are administered together, or that both drugs are frequently taken together for convenience. As one or both drugs are typically already approved, the 505(b)(2) pathway is the obvious choice for approval of many drug-drug combination products. We have […]
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Faster Approval of Combination Drug Products Via the 505(b)(2) Pathway

On April 11 and 12, 2016, Ken Phelps, President and CEO of Camargo Pharmaceutical Services, spoke at the 505(b)(2) Forum and CTrials Conference in Tel Aviv, Israel, on the topic of 505(b)(2) Combination Drug Products. What is attractive about the 505(b)(2) regulatory pathway, and specifically, why is it so beneficial for approval of combination products? […]
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Product Ideation: Identifying Your Optimal Drug Development Candidate

It’s a familiar story: Pharmaceutical Company X spends years developing Product Y only to discover at a crucial point in the process that Product Y will not succeed. The result is often millions of dollars and development years wasted. But could drug development failure be prevented? Oftentimes, drug development failure can be attributed to misalignment […]
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Pediatric Applicability or Not–This Revised Guidance Is for You

Since 1994, the statutory and regulatory requirements for drug product labeling for pediatric populations have been evolving. The FDA Modernization Act of 1997 (FDAMA) contained incentives for conducting pediatric studies on drugs that had exclusivity or patent protection. In 2003, the Pediatric Research Equity Act (PREA) was signed into law requiring, for certain drugs, sponsors […]
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Is a Reference Listed Drug Mandatory in the 505(b)(2) Pathway?

If you are a frequent reader of our blog, you know that the 505(b)(2) pathway can facilitate a cheaper, faster drug approval route. This is accomplished by relying on: 1) safety and efficacy data from published literature and/or 2) the agency’s assessment of safety and efficacy of a FDA-approved product, known as the “reference listed […]
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Additional 505(b)(2) Benefits: Selective Safety Data Collection

  Last month CDER/CBER released a short, final guidance, “Determining the Extent of Safety Data Collection Needed in Late Stage Premarket and Postapproval Clinical Investigations.” (CDER/CBER, 2016) While brief, the guidance could provide a significant reduction in safety data collection for NDA sponsors. This could be especially true for sponsors using the 505(b)(2) regulatory pathway. […]
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How Much Is a First Cycle Review ANDA Approval Worth to You?

At the recent GPhA meeting in Orlando, Florida, Dr. Kathleen Uhl from the FDA Office of Generic Drugs (OGD) spoke about the quality of Abbreviated New Drug Application (ANDA) submissions and highlighted the detriments of a poor quality submission. All would agree that a poor quality submission is costly for drug product development. Dr. Uhl […]
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Return on Investment for 505(b)(2) Products: Is an “AB” Rating Possible?

Return on Investment for 505(b)(2) Products: Is an “AB” Rating Possible? As new generic product options become more scarce and the competition more fierce, generic manufacturers have recently become very interested in the 505(b)(2) regulatory pathway. The benefits of the 505(b)(2) pathway are well known, such as a potentially lower cost and accelerated path to […]
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2015 505(b)(2) NDA Approvals

2015 was a big year for FDA, with 45 novel new therapies approved — much higher than 28, the average number approved in the past decade. Of the 45 approved in 2015, how many were approved through the 505(b)(2) pathway? And how does the number of approvals through 505(b)(2) compare with previous years? The results […]
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3-Year Exclusivity May Not Be Worth as Much as You Think

It is a widely held tenet that market exclusivity is essential for the successful launch of a new drug. But is this always the case? For products approved through the FDA 505(b)(2) pathway, is pursuing the 3-year period of exclusivity available through Hatch-Waxman always necessary? Or is it possible that a product may succeed without […]
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Drug Development Question? Here’s how to communicate with the FDA!

Earlier this month FDA (CDER and CBER) issued a new draft guidance, Best Practices for Communication Between IND Sponsors and FDA During Drug Development. Based on Camargo’s frequent communications with the Agency during product development, the guidance does not present any profound or significant changes in how they conduct and or prefer communicating with IND […]
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New Nonclinical Guidance for 505(b)(2) Products: No Cause for Alarm

On October 15, 2015, the United States Food and Drug Administration (FDA) issued a new guidance for industry and review staff with more uniform recommendations for the nonclinical safety evaluation of approved drug substances that are reformulated or in which a new route of administration is proposed for a previously approved drug product. These products, […]
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The Winding Path to Derisking Formulation Changes

Originally posted by AAPS Blog 10/7/15 If you are a researcher involved in drug development, you are familiar with the inevitability of change and the need to manage and understand it throughout the development process. No question, it is the status quo, and it must be planned for and managed. As researchers, we know where […]
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Encounters of the Third Sector

As I walked the floor at the International Generic Pharmaceutical Alliance (IGPA) conference in Toronto, I couldn’t help but feel a little gratified. Companies that were once disavowing the 505(b)(2) approval pathway are now driving the conversation. Investors that once shied away from 505(b)(2) products due to their unknown reputations despite the relatively low risks […]
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Enforcement Activities: FDA removes unapproved prescription ear drops

For years FDA has threatened to remove unapproved products (so-called DESI products) from the marketplace. Recently, the FDA took enforcement action against  several unapproved prescription ear drops.  What products will be next?  DESI producers can use the 505(b)(2) pathway to avoid such actions on their products. Let’s take a look at the recent action and show an example of  […]
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Examining the Amarin VASCEPA Saga

The headlines and newscasts reported Amarin’s success in wining off-label promotion, but behind the scenes, another noteworthy action took place – in a very rare action, the FDA rescinded a special protocol assessment (SPA) that would have enabled Amarin to promote the new indication. In this post, we’ll examine the reasons for FDA’s action and […]
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Quality Metrics – Coming Soon to Your Manufacturing Facility

In the July 28th, Federal Register, (Under “Meetings”) FDA announced the availability of the draft guidance “Request for Quality Metrics-Guidance for Industry.” Although there are “requests” for quality metrics in the guidance, the bulk of the document tells industry what quality metrics they will be reporting, at least initially. FDA primarily cites Sections 706 and […]
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Use of Extrusion-Enabled Pharmaceutical Processes in Drug Development via a Streamlined Regulatory Pathway

Extrusion-enabled pharmaceutical processing (E2P2) has long been employed in pharmaceutical development (Drug Development and Industrial Pharmacy. 33:909-926,1043-1057 (2007)). Reasons for utilizing E2P2 include: Enhancing physico-chemical properties of APIs such as solubility and stability Modifying in vivo release of a drug to enhance bioavailability and clinical efficacy Facilitating development and manufacture of fixed combination drug products […]
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Active Ingredients vs. Active Moieties – Perplexity of Understanding the Relationship or Distinction

Recently a federal district court spotlighted FDA’s apparently inconsistent definitions of what constitutes an “active ingredient (AI)”  in rejecting the Agency’s rationale for denying Amarin Pharmaceuticals Inc.’s fish oil capsule Vascepa (icosapent ethyl) (NDA 202057 approved July 26, 2012), request for 5 years of market exclusivity as a New Chemical Entity (NCE).  On Feb. 21, […]
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To List or Not to List – That is the Question

A 505(b)(2) may rely on the FDA’s previous findings of safety and efficacy  of an approved drug product. It is possible to rely on more than one approved drug product.  It is also possible that a 505(b)(2) applicant does not have to rely on any approved drug. The correct choice of listed drug product may allow  […]
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Pediatrics – What are the appropriate age ranges?

As we have noted in this blog previously, under the Pediatric Research Equity Act (PREA), all new drug applications for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration are required to contain an assessment of the safety and effectiveness of the product for the claimed indication(s) […]
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Priority Review Vouchers are a Big Carrot for Hungry Companies

Priority review vouchers (PRVs), which are fast-becoming a powerful incentive for drug companies, were originally based on a publication (Ridley et al. 2006) from a group at Fuqua School of Business at Duke University in NC. The idea behind PRVs was that developers of treatments for neglected infectious diseases (and later, rare pediatric diseases) would […]
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PhRMA and GPhA team up (!) to offer their EAR proposal to solve generic safety labeling issue

FDA held a public hearing this past Friday (27March2015) to air their proposal that generic companies be responsible for updating their labeling whenever ‘new safety information’* is available. Generic companies argue against such a proposal because it would lead to confusion about safety warnings if every multisource product has different labeling (coincidentally, and privately,  they […]
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Opportunities in Orphan Drug Development for Investors, Pharma and CROs

Orphan drugs, defined in the Orphan Drug Act as drugs developed to treat rare diseases that affect fewer than 200,000 people in the U.S., have begun to make their mark for patients and drug companies. As the number of orphan drugs has increased over the past 30 years, many patients with rare diseases have benefited […]
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REMS/ETASU and Safe Use in Bioequivalence trials

We’ve previously commented regarding the predilection of RLD holders whose product approvals include a Risk Evaluation and Mitigation Strategy (REMS) and Elements To Assure Safe Use (ETASU) to use the REMS/ETASU as a barrier to entry for generic completion.  Specifically, the RLD holder will refuse to sell their product to the prospective generic manufacturers, who […]
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MAPPing out the timing of a Complete Response submission

A type of FDA document which sometimes slides past under the radar is  MAPP, that is, Manual of Policies and Procedures.  These are actually internal FDA documents which are generally analogous to the SOPs FDA requires that industry have and follow.  However, by virtue of the various requirements for transparency placed on FDA, they are […]
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