The Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee met on September 28th to discuss the new drug application (NDA 200896) for Ataluren®.
The Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee met on September 28th to discuss the new drug application (NDA 200896) for Ataluren®. Ataluren, sponsored by PTC Therapeutics, Inc, is a novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation in the dystrophin gene. Dystrophinopathies are a group of conditions that generally cause muscle weakness.
The FDA’s conclusion was that the studies had significant technical and design issues resulting in data that cannot be interpreted and thus, the analysis conducted by the applicant is not supportive of an inverted‐U shaped dose‐response for Ataluren. The panel was asked to vote on the best interpretation of the information presented regarding the use of Ataluren for the treatment of dystrophinopathies resulting from nonsense mutations in the dystrophin gene. Specifically, the committee was asked whether the data were A) not effective, B) inconclusive, or C) sufficient to conclude that Ataluren is effective.
The results from the panel were A: 0 B: 10 C: 1, with the majority of the committee voting that the data was inconclusive, and more work would be needed to establish whether Ataluren is effective. These members also generally agreed that the testimonies from the public appeared compelling and should encourage the applicant to continue working on the trials. The one committee member who voted that the data are sufficient to conclude that Ataluren is effective, noted that his vote was based on written reports by clinicians on their observations of patients who have shown positive results with Ataluren and on the public testimonies describing patients who experienced reversal of symptoms when Ataluren was removed and improvement when it was reintroduced.
The role of the PCNS advisory committee is to provide recommendations to the FDA, in which the agency will then take into consideration when determining its final approval decision. The panel’s vote reduces the chance the drug will be approved as the FDA generally follows the advice of its advisors. However, the FDA is generally supportive of PTC’s ongoing evaluation of the efficacy of Ataluren based on the exploratory findings. The Agency believes that exploratory analyses from negative trials can generate hypotheses and such hypotheses need to be prospectively tested.
ISS has almost two decades of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact firstname.lastname@example.org
Do you have an upcoming Advisory Committee Meeting?
Over the past few years, the number of ex-US manufacturers submitting a Class II exempt 510k device registration has increased substantially. For companies with medical devices that are approved and successful in a foreign market, the logical next step is to introduce their product into the US, which has one of the most lucrative healthcare markets in the world. Read more
FDA advisory committee meetings are important regulatory events for many manufacturers on the path to marketing approval. These meetings can quite literally result in the approval or rejection of a product based on the panel’s vote. Read more
One of the main challenges our clients face is determining which regulatory pathway is best for their products. For example, what if an OTC product has a Monograph active ingredient but that ingredient is present at a different amount than the permitted amount? Or what if the label claims a novel indication not specified in a Monograph? If you are debating pursuit of either regulatory pathway for your OTC drug, it is critical to consider cost, timeline, and label claims to ensure your products has a unique stance in today’s market. Read more
US distributors and agents trying to import rapid antibody tests for coronavirus face a number of obstacles. The good news is that FDA has opened up the Emergency Use Authorization (EUA) program to include SARS-CoV-2 (the virus) and COVID-19 (the disease), expediting time to market. Read more
The journey to scientific and commercial success is often complex and always critical, if you are looking for an expert partner to help steer you to confident solutions, contact us todayContact us