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Orphan Drug Articles and Insights

PDUFA VII: Implications for Developers of Cell and Gene Therapies

One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of the advancements included in PDUFA

In the News: Regulatory and Development Updates

Rare Disease Clinical Trials Most Often Terminated Due to Regulatory and Recruitment Issues The difficulty associated with successfully completing rare disease clinical trials is well-known. Most people believe that recruitment is the primary reason because, by definition, the number of potential trial participants is extremely

FDA Designations for Rare Disease Products, Part 1

Comparing Orphan Drug, Rare Pediatric Disease, and Humanitarian Use Device Designations The FDA offers sponsors a variety of special programs to incentivize development of therapies to treat unmet patient needs, including fast track, breakthrough therapy, and qualified infectious disease product designations. This blog post initiates a four-part series

Rare and Orphan Drug Development in the EU

A pharmaceutical company is granted orphan designation for a medicine that treats Hunter Syndrome. The company goes on to receive marketing approval for the medicine. The approved medicine is administered through intravenous infusion. This route of administration, while effective for some, does not address the

Scope of Orphan Drug Exclusivity – How Broad is Broad?

It has recently been reported that drugmakers have argued against broad orphan exclusivity for Eagle Pharmaceuticals, Inc.’s Bendeka® product. This was in response to the FDA’s invitation to applicants of certain products containing bendamustine to share how broad of a scope they believe Eagle’s seven-year

How to Get Orphan Status for 505(b)(2) Drugs

2017 was a big year for orphan designations and approvals. Sixteen of the sixty-three (25%) products approved via the 505(b)(2) pathway in 2017 received orphan designation. Importantly, more than half of the approved 505(b)(2) products with orphan designation received seven years of orphan exclusivity. That’s

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

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

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.

Orphan Designation without Exclusivity: Court asked to decide

Yesterday, Depomed filed suit against the FDA requesting the Court to order FDA to grant their product Gralise (gabapentin) seven years of exclusivity since it was granted Orphan status; upon approval, Gralise was granted three years of exclusivity. Depomed licensed the product to Solvay which became

505(b)(2) Patent & Marketing Exclusivity

IP attorney Stephen Albainy-Jenai and I just concluded a webinar hosted by DIA entitled 505(b)(2) Patent & Exclusivity.  23 different companies attended, showing the increasing interest in 505(b)(2) issues.  Earlier this year, DIA hosted my overview of the 505(b)(2) drug development process where the attendees had many questions asking