Zevra Therapeutics

Zevra Therapeutics published long-term data showing MIPLYFFA (arimoclomol) sustained disease progression reduction for at least 5 years in Niemann-Pick disease type C patients.

Bavarian Nordic announced the sale of its Priority Review Voucher for $160 million, awarded following FDA approval of its chikungunya vaccine VIMKUNYA in February 2025.

The median annual list price for newly launched pharmaceuticals in the U.S. has more than doubled to $370,000 in 2024 compared to $180,000 in 2021, according to a Reuters analysis.

• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator. • The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments. • Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.

DelveInsight's latest report reveals 8+ companies developing 10+ therapies for hypersomnia, with Avadel's Lumryz, Zevra's KP1077, and Alkermes' ALKS 2680 among the key candidates advancing through clinical trials.

Priority Review Vouchers (PRVs) have surged in value to $150-158 million in recent months, up from the typical $100 million, following Congress's failure to reauthorize the rare pediatric disease program.

The FDA's rare pediatric disease Priority Review Voucher program expired in December 2023, despite unanimous congressional support for renewal, leaving biotech companies unable to receive new vouchers worth approximately $150 million.

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

• Zevra Therapeutics has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million, with the transaction expected to close within 30-45 days. • The PRV was awarded following FDA approval of MIPLYFFA, a treatment for neurological manifestations of Niemann-Pick disease type C in patients aged 2 and older. • The deal is subject to standard closing conditions, including Hart-Scott Rodino Antitrust Improvements Act waiting period expiration, with Cantor Fitzgerald serving as financial advisor.

The FDA has awarded $17.2 million in clinical trial grants for seven rare disease treatments, including therapies for Cushing's syndrome, blood disorders, cancer, and an inherited eye disease.