Therapeutic Areas Resources

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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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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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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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. From the House Committee on Energy and Commerce’s bill summary/discussion […]
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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 Guidance in effect, a drug product will be eligible for […]
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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). In many cases, some portion of the limited efficacy is […]
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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 as soon as January 2015. This approval has been a […]
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PREA – Pediatric Plan Timing Changed by PDUFA V

The Food and Drug Administration Safety and Innovation Act (FDASIA; also known as PDUFA V), signed into law on July 9, 2012, contains amendments to the Pediatric Research Equity Act (PREA) that specifically detail the timing of the submission of a Pediatric Study Plan (PSP). In order to direct greater adherence to the PREA requirements before […]
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FDA Goes Against Advisory Committee and Approves Low Dose Paroxentine for Hot Flashes

On 6/30/2013, FDA approved Noven Pharmaceuticals Brisdelle (paroxetine) for the treatment of vasomotor symptoms (hot flashes).  This 505(b)(2)  approval is notable since it is the first non-hormonal product product approved for this indication. But from a regulatory point of view, it is even more interesting – FDA went against a 10-4 Advisory Committee vote against […]
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FDA Rejects Depomed’s Gabapentin for Menopausal Symptoms

FDA concurred with its Advisory Committee’s recommendations and has turned down Depomed’s application seeking a new indication for gabapentin.  Depomed has sought approval to use the drug in the treatment of menopausal symptoms – ‘hot flashes’. We reviewed the Advisory Committee’s deliberations in this blog this past April.  The drug failed to show superiority over placebo.  […]
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Advisory Committee Cool on Non-Steroid Hot Flash Treatments

The Camargo team got its start in the 505(b)(2) process at Duramed Pharmaceuticals with the 1990’s development and approval of Cenestin (synthetic conjugated estrogens, A).  The product was approved based on a Phase 3 clinical study demonstrating the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause –   (known as ‘hot flashes’).  For decades, estrogens have […]
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AB Rated 505(b)(2)’s

Can you have an “AB” rated 505(b)(2)?  Yes, as well as other Therapeutic Equivalent (TE) codes that are most often associated with the TE codes for generics in the Orange Book. Several years ago when I was speaking about the potential products that qualified under 505(b)(2) I had a line in a PowerPoint slide for […]
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Ophthalmics: 21 CFR 314 94(a)(9)(iv) no longer applies

Who would have guessed that 21 CFR 314.94(a)(9)(iv) no longer applies to ophthalmics?  You wouldn’t generally have expected it to just be cancelled – normally FDA must go through notice and comment, but apparently the FDA can make a regulation disappear by decree. 21 CFR 314(a)(9)(iv) states: (iv)Inactive ingredient changes permitted in drug products intended […]
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What is the Pediatric Drug Development Approach for Rare Diseases and Orphan Drugs?

As part of the PDUFA V reauthorization discussions, the FDA and industry are talking about better approaches to rare disease drug development and orphan drugs. Public interests and Congress have mandated that FDA develop new guidances on the required studies needed for NDA approval. In these discussions, participants refer to the “pediatric drug development approach” […]
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Contrave® Rejection: The Long and the (Too) Short of it

On February 1st Orexigen(R) Therapeutics, Inc. and Takeda Pharmaceutical Company Limited (Takeda) announced that the FDA issued a complete response letter dated January 31, 2011 regarding the New Drug Application for Contrave® (naltrexone HCl/bupropion HCl) extended-release tablets for the treatment of obesity, including weight loss and maintenance of weight loss (http://ir.orexigen.com/phoenix.zhtml?c=207034&p=irol-newsArticle&ID=1522207&highlight=). In the letter, FDA […]
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Nuedexta® – Smart Pharmacology to Treat a Unique Disorder

The FDA has approved NuedextaÃ’ (Avanir Pharmaceuticals Inc.), a drug that curbs involuntary and uncontrolled crying and laughing episodes (known as pseudobulbar affect (PBA)) that are experienced by patients with some neurological disorders. Nuedexta is the first drug to be approved to treat patients with these symptoms. Nuedexta is a combination product containing dextromethorphan hydrobromide […]
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The Skinny On a Potential New Treatment of Obesity

Orexigen® Therapeutics is developing a new fixed dose sustained-release (SR) combination of naltrexone and bupropion for the treatment of obesity. The rationale behind the two active ingredients is stated to be: Bupropion acts on the weight control circuit by stimulating the POMC neuron. Naltrexone prevents inhibition of POMC neurons by blocking the action of β-endorphin […]
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Target Product Profile

Today’s blog courtesy of Lynn Gold, Ph.D. Camargo’s VP of CMC. In any project development program an understanding of the program goal is critical to finding the shortest path to the final result. Generation of a Target Product Profile early in a development program facilitates reaching the goal of a marketed drug product. It provides […]
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Pediatric Assessments in Drug Development: Timing in Europe vs. US

As we have noted in this blog on several occasions, under the Pediatric Research Equity Act (PREA) (21 U.S.C. 355c), 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 […]
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A New Paradigm for the Development of Drugs for Type 2 Diabetes

Due to Camargo’s on-going client projects in this area, our chief medical officer, Dr. Sam Kaba recently attended a DIA-sponsored conference in Washington, D.C. entitled Cardiovascular Safety and Development of Type 2 Diabetes Mellitus Medications: Current State of the Art and Opportunities to Advance the Science. His report: The FDA Guidance for Industry on Evaluating […]
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505(b)(2) Program: BTG’s Paxclitaxel Gel for Oesophageal Cancer

Media is reporting that BTG’s Phase IIa study of a novel gel formulation of paxclitaxel showed tumor reduction in 70% of patients.  In effect, BTG is taking a known agent and directly targeting the affected tissues;  BTG is also reportedly in later phase trials looking at this gel product in brain cancer – placing the […]
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PREA and 505(b)(2)

Under the Pediatric Research Equity Act (PREA) (21 U.S.C. 355c), all applications for new active ingredients, new indications, new dosage forms, new dosing regimens, or new routes of administration are required to contain an assessment of the safety and effectiveness of the product for the claimed indication(s) in pediatric patients unless this requirement in waived, […]
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Suicidal behavior: Ignore therapeutic area at your own risk

We hope a common theme emerges from this blog and our public interactions: it is vital when planning 505(b)(2) drug development to look at the entire therapeutic class to learn what studies are needed for approval of the drug .  Not including the class in your background of public information can be suicidal. FDA regulates […]
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