Zevra Therapeutics

XOMA Royalty reports Q3 2024 financials: $7.2M income, FDA approval for Zevra’s MIPLYFFA™ (arimoclomol), $15M royalty monetization with Twist Bioscience, $9.9M cash receipts in Q3, $42.3M YTD.

Azitra, Inc. to present at 2024 Maxim Healthcare Virtual Summit, with COO Travis Whitfill participating in the rare disease panel on October 17, discussing innovative precision dermatology approaches.

Azitra, Inc. to present at the 2024 Maxim Healthcare Virtual Summit on October 17, 2024, at 2:00 p.m. ET, as part of the rare disease panel, alongside Quoin Pharmaceuticals, Rezolute, and Zevra Therapeutics.

FDA approves Miplyffa (arimoclomol) for treating NPC in adults and children aged 2+ in combination with miglustat, based on a 12-month trial showing slower disease progression. Common side effects include upper respiratory tract infection, diarrhea, and weight loss.

In September 2024, the FDA made several key decisions, including the clearance of Cortechs.ai’s NeuroQuant 5.0 software for advanced MRI lesion segmentation, the breakthrough therapy designation for Simcere Pharmaceuticals’ edaravone dexborneol for acute ischemic stroke, the acceptance of Axsome Therapeutics’ resubmitted NDA for AXS-07 for acute migraine, the support for α-synuclein seed amplification assay biomarker in Parkinson disease trials, the approval of subcutaneous ocrelizumab for multiple sclerosis, the approval of Zevra Therapeutics’ arimoclomol for Niemann-Pick disease type C, and the approval of IntraBio’s levacetylleucine as a stand-alone treatment for NPC.

September 2024 FDA updates include approvals for Embecta's tubeless patch pump for diabetes, Travere Therapeutics' sparsentan for IgA nephropathy, Johnson & Johnson's guselkumab for ulcerative colitis, and the first over-the-counter hearing aid software device. Other approvals cover treatments for conditions like chronic rhinosinusitis with nasal polyps, atopic dermatitis, eosinophilic granulomatosis with polyangiitis, Niemann-Pick disease type C, and schizophrenia. The FDA also issued warnings and accepted NDAs for various treatments, including delgocitinib cream for chronic hand eczema and roflumilast foam for psoriasis.

The FDA approved two drugs, Miplyffa and Aqneursa, for treating Niemann-Pick disease type C within a week, offering the first treatments for this fatal disorder. Miplyffa, a combination therapy with Zavesca, demonstrated efficacy in halting disease progression, while Aqneursa, a standalone therapy, showed significant neurological improvements. Both companies launched support programs to aid patient access to these treatments.

FDA approves Zevra Therapeutics' Miplyffa (arimoclomol) for treating Niemann-Pick disease, type C (NPC) in adults and children aged 2 and older, in combination with miglustat. The approval is based on a 12-month trial showing slower disease progression with Miplyffa, though it caused common side effects like upper respiratory tract infection, diarrhea, and decreased weight.

FDA approves IntraBio's Aqneursa for NPC neurological symptoms in adults and pediatric patients, following IB1001-301 study showing significant improvements within 12 weeks.