Zevra Therapeutics

Zevra's MIPLYFFA maintains market confidence despite AQNEURSA competition, with strong stock returns and revenue growth. MIPLYFFA's early success, including 90 prescriptions and 30% reimbursement approval, projects $230M U.S. sales by 2028. Zevra's pipeline includes KP-1077 and Celiprolol, and a Priority Review Voucher could fetch $110M+. OLPRUVA underperforms but shows potential.

2024 saw significant medical advances, including FDA approval of Orlynvah for resistant UTIs, Cobenfy for schizophrenia, lenacapavir for HIV prevention, and Miplyffa/Aqneursa for Niemann-Pick disease type C. Elinzanetant showed promise for hot flashes, and future innovations include potential non-opioid pain treatments, improved flu vaccines, and gene therapy for vision restoration.

Zevra Therapeutics launched Miplyffa, the first treatment for Niemann-Pick disease type C, available since November at over $100,000 per month. The drug, used with miglustat, has seen early adoption success and aims to increase disease diagnosis. Zevra plans to expand Miplyffa to Europe in 2025 and continues to develop other treatments for rare conditions.

Zevra Therapeutics announces commercial availability of MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick Disease Type C (NPC), used with miglustat to treat neurological symptoms in adults and children. AmplifyAssist™ supports patients in accessing this therapy.

Zevra Therapeutics announced FDA-approved MIPLYFFA™ (arimoclomol) for Niemann-Pick disease type C (NPC) is now available, prescribed with miglustat for neurological symptoms in NPC patients aged two and older. Early demand surpassed expectations, with the AmplifyAssist™ program offering support to patients and caregivers. MIPLYFFA increases transcription factors EB (TFEB) and E3 (TFE3) activation, upregulating genes associated with lysosomal expression and regulation (CLEAR), and demonstrated a reduction in unesterified cholesterol in lysosomes of human NPC fibroblasts. The pivotal phase 3 trial showed a halt in disease progression over a one-year period. MIPLYFFA received Breakthrough Therapy, Rare Pediatric Disease, Orphan Drug, and Fast Track designations from the FDA, and Orphan Medicinal Product designation from the European Medicines Agency (EMA) for NPC treatment.

Jason McCarthy of Maxim Group maintains Buy rating on Zevra Therapeutics (ZVRA) with a $25.00 price target, citing strong financials, FDA approval of Miplyffa for NPC, and commercial readiness of Miplyffa and Olpruva. ZVRA's stock has increased by 88.12% in six months.

Zevra Therapeutics is enrolling patients in a Phase 3 trial, DiSCOVER, testing celiprolol, a beta blocker for vascular Ehlers-Danlos syndrome (vEDS) caused by COL3A1 mutations. The trial aims to reduce medical emergencies and is under a special protocol assessment agreement with the FDA. Celiprolol, previously tested in France, showed reduced risk of blood vessel ruptures. DiSCOVER plans to enroll 150 vEDS patients, with the primary goal of reducing clinical events requiring medical intervention over 40 months.

William Blair analyst Sami Corwin maintains a Buy rating on PTC Therapeutics (PTCT) stock due to the FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, and the company's proactive commercial launch preparations. Corwin's rating is further supported by the priority review voucher and promising clinical trial results.

Zevra Therapeutics reports Q3 2024 financial results and FDA approval of MIPLYFFA™, ending the quarter with $95.5 million in cash, cash equivalents, and investments.

Zevra Therapeutics reports Q3 2024 financial results, including FDA approval of MIPLYFFA™ for NPC, a rare pediatric disease Priority Review Voucher, and a cash position of $95.5 million.