FDA committee endorses Zevra Therapeutics’ arimoclomol as a treatment for Niemann-Pick Disease Type C

In a notable advancement for rare disease treatment, Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) has received a significant endorsement from the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC). The committee voted 11 to 5 in favour of arimoclomol, affirming that the data supports its effectiveness in treating Niemann-Pick Disease Type C (NPC).

Key details of the advisory committee’s decision

Zevra Therapeutics expressed strong satisfaction with the committee’s vote. Neil F. McFarlane, President and Chief Executive Officer of Zevra Therapeutics, commented on the positive outcome, noting the extensive clinical data that supports arimoclomol. This data includes pivotal trial results, long-term data from the open-label extension study, and information from expanded access programmes. McFarlane remained optimistic about the continued approval path for arimoclomol.

FDA advisory committee’s review process

The FDA Genetic Metabolic Diseases Advisory Committee, composed of experts in medical genetics, inborn errors of metabolism, and related fields, assessed arimoclomol’s benefits and risks. The committee reviewed data presented at the 45th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD) and considered input from independent experts, NPC patients, and patient advocacy groups. While the committee’s recommendation is influential, it is not binding. The FDA will incorporate this feedback as it completes its independent review of the arimoclomol New Drug Application (NDA). The Prescription Drug User Fee Act (PDUFA) action date for arimoclomol is set for September 21, 2024.

Understanding Niemann-Pick Disease Type C

Niemann-Pick Disease Type C is a rare, progressive lysosomal storage disorder that affects the body’s ability to transport cholesterol and other lipids within cells. This leads to their accumulation in various tissues, including the brain. NPC results from mutations in the NPC1 or NPC2 genes, essential for producing lysosomal proteins. Both children and adults can be affected, with the disease causing severe physical and cognitive impairments. The progression of NPC is typically irreversible and can be fatal within months or years.

Arimoclomol’s role in rare disease treatment

Arimoclomol, an investigational drug product candidate from Zevra Therapeutics, has received multiple designations from the FDA, including Orphan Drug, Fast Track, Breakthrough Therapy, and Rare Pediatric Disease designations. The drug has also been granted Orphan Medicinal Product designation by the European Medicines Agency (EMA). The FDA has accepted the resubmission of arimoclomol’s NDA, with a user fee goal action date (PDUFA date) of September 21, 2024. Arimoclomol aims to address the significant unmet needs of NPC patients by potentially slowing disease progression and improving their quality of life.

Zevra Therapeutics: Leading the fight against rare diseases

Zevra Therapeutics is dedicated to developing transformative therapies for rare diseases with few or no existing treatments. The company’s approach combines scientific innovation with a focus on patient needs to overcome complex drug development challenges. Zevra’s commitment to addressing rare diseases is exemplified by its efforts with arimoclomol, showcasing its role in advancing treatment options for conditions like Niemann-Pick Disease Type C.

What was the outcome of the FDA committee’s review of arimoclomol?

The FDA Genetic Metabolic Diseases Advisory Committee voted in favor of arimoclomol, indicating that the presented data supports its efficacy for treating Niemann-Pick Disease Type C. This positive vote is a crucial step in the drug’s approval process.

What does this mean for Niemann-Pick Disease Type C patients?

The endorsement by the FDA advisory committee suggests that arimoclomol could soon become an available treatment option, offering hope to patients suffering from this rare and often debilitating condition. It promises to potentially slow disease progression and improve quality of life for affected individuals.

What are the next steps following the advisory committee’s recommendation?

While the advisory committee’s recommendation is a positive development, it is not binding. The final decision rests with the FDA, which will consider this recommendation and complete its review of the arimoclomol New Drug Application (NDA). The decision is expected by the Prescription Drug User Fee Act (PDUFA) action date on September 21, 2024.