US FDA Approves Zevra’s Treatment for Rare Genetic Disease, Reports Reuters

By Sriparna Roy

The U.S. Food and Drug Administration has granted approval for Zevra Therapeutics’ drug, marking it as the first treatment available for a rare and lethal genetic disorder. This announcement was made by the health regulator on Friday.

Zevra Therapeutics has been working for several years to bring this drug to market after the FDA had previously turned down its application and extended the review process.

The oral medication, known as Miplyffa, has now received approval for the treatment of Niemann-Pick disease type C, a rare genetic condition that impacts the nervous system and other bodily organs. Those diagnosed with this disease typically have a life expectancy of approximately 13 years.

"The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering," stated Janet Maynard, a director at the FDA’s Center for Drug Evaluation and Research.

Miplyffa, when used alongside miglustat, known as Zavesca, has been authorized to alleviate neurological symptoms related to NPC in both adults and children aged 2 years and older. The drug is anticipated to be available in the U.S. within 8 to 12 weeks, according to Zevra.

The company has not yet responded to inquiries regarding the pricing of the drug. Analyst Oren Livnat from H.C. Wainwright predicts the average net price to be between $500,000 and $600,000 annually, estimating peak sales in the U.S. could reach around $250 million.

Miplyffa carries warnings about potential hypersensitivity reactions, which may include hives and angioedema, a condition characterized by swelling beneath the skin.

Zevra gained access to Miplyffa through its acquisition of Orphazyme, the drug’s original developer, in 2022. Following the approval news, shares of Zevra rose by more than 5%, reaching $8.43 in midday trading.