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505(b)(2) Development Articles and Insights

Repurposing Drugs As an Answer to the COVID-19 Crisis

Hydroxychloroquine. The world rallied around this old drug as a potential treatment of COVID-19 patients. The pharma industry, independent researchers, and governments rushed to conduct trials to see if hydroxychloroquine is safe and effective at any stage of this invasive virus. Though it has now

Three Keys to Preparing Effective Pre-IND Meeting Questions

Asking the appropriate questions during a Pre-IND meeting with the FDA is a critical step in planning a development program. A Pre-Investigational New Drug Application (Pre-IND) meeting can be a valuable component in planning a development program. For companies that have not previously interacted with

2019 505(b)(2) NDA Approvals in Review

In 2019, CDER approved 64 NDAs that used the 505(b)(2) pathway, representing important advances in patient care across a wide range of therapeutic areas. The number of 2019 NDA approvals that used the 505(b)(2) pathway fell from 75 in 2018 to 64 in 2019 (Figure 1), in

ANDAs Prioritized to Combat Opioid Crisis

As part of nationwide efforts to address the opioid crisis, FDA is taking a closer look at the development of critical medications that combat overdose deaths. Most recently, FDA announced its intention to prioritize review of ANDAs for drug products indicated for emergency use in

505(b)(2) or ANDA: Which Pathway is Right for Your Product?

Last week, FDA published new guidance, Determining Whether to Submit an ANDA or a 505(b)(2) Application (CDER, 2019), to help Sponsors select the best abbreviated approval pathway for their product. While the guidance does not provide any monumental new initiatives, policies or approaches, here are four key

2017 – A Great Year for Generics – Yes or No?

Generic Firms Looking for Revenue with 505(b)(2) AAM’s Annual Conference is over and most of the generic companies are glad 2017 is over, too. Despite media reports that 2017 was the best year ever for generics, only 3 companies of the top 30 companies reported

505(b)(2) Success Requires More Than Just Science

At the JP Morgan Conference/Biotech Showcase this year, we encountered a record number of companies engaged in 505(b)(2) development. We also had meetings with investors and buyers with their checkbooks open to finance or acquire the more than 80 products we have in development. Unfortunately,

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

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

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

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

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)

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

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

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

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

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

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

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?

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

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

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

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

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

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

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

Extending Exclusivity: How Long Will It Really Last?

Last week at the Generic Pharmaceutical Association (GPhA) Annual Meeting, the 21st Century Cures Act, a proposed bill with bipartisan support, was a topic of discussion. Specifically, subtitle L—Dormant Therapies, which would offer 15 years of exclusivity for drugs and biologics approved as dormant therapies.

2014 505(b)(2) NDA Approvals

2014 drug approvals seem to have rebounded somewhat from the past year. In his annual CDER New Drug Review Update, FDA’s John Jenkins cited 35 NME NDA and BLA approvals (calculated through December 3, 2014), up from 27 in 2013 (see chart below). These approvals

Therapeutic Equivalence Ratings Under 505(b)(2)

The FDA listing of therapeutic equivalence (TE) ratings can be a murky area for products approved under 505(b)(2) applications. The concept of TE ratings emerged from FDA regulations for generics and revolve around the announcement that the FDA would publish a current listing of all

5-Year Exclusivity for Certain Fixed-Combination Drugs with an NCE

The FDA recently posted new Guidance on its website awarding certain fixed-combination drug products (fixed-combinations) 5-year new chemical entity (NCE) exclusivity. While the Agency held previously that these products were ineligible for NCE exclusivity (5 years) if one component is already approved, with this new

Alkermes Prodrug for Treatment of Multiple Sclerosis: NCE?

The Food and Drug Administration (FDA) began requiring drug efficacy, in addition to safety, for approval in 1962 based on the Kefauver-Harris Amendment. Despite this requirement, many drugs that have been approved by FDA have limited efficacy (eg, drugs that treat cancer or Alzheimer’s disease).

Paper Submissions: Going, going…away

In order to fulfill a requirement specified in Section 745A(a) of the Food and Drug Administration Safety and Innovation Act (aka FDASIA), FDA recently issued a draft guidance directing mandatory use of electronic filing and formatting for most regulatory submissions which currently can still be submitted

MannKind Breathes Easier – Inhaled Insulin Finally Approved

MannKind’s Afrezza Receives FDA Approval In June of this year, MannKind Corporation announced that they received FDA approval for Afrezza®, their rapid-acting inhaled insulin product. MannKind is currently working to identify a pharma partner to manufacture and distribute Afrezza, and the product could be available

2013 505(b)(2) NDA Approvals

2013 appears to be a challenging year for FDA NDA approvals.  FDA's John Jenkins reviewed the  NME NDA and BLA approvals through November 2013, showing that 25 such products were approved (see chart below).  These 25 approvals are 505(b)(1)NDA and BLA's.  Generally, this performance was seen as

Expensive, cheap, value? CMO costs.

Contract manufacturing organization (CMO) A bids $600,000.  CMO B bids $450,000.  Which bid is cheaper? Assume for the moment that both CMOs provide equal service parameters: skill, capacity, timelines, regulatory history, experience, etc. and we are judging both based only on price. No, this is a not trick question. 

ANDA but No NDA – What to Rely on?

Camargo participates in 2-5 PIND meetings each month and one thing we notice in the FDA minutes is that the boilerplate answer to 'does the Agency agree this ..... is appropriate for filing under 505(b)(2)?' keeps getting longer.  Recently, the Agency (or, at least, some divisions) is