Hunter Syndrome Pipeline
Mar 30, 2026
Denali’s AVLAYAH Win for Hunter Syndrome Marks Turning Point for Rare Disease Innovation
Summary AVLAYAH (tividenofusp alfa‑eknm) has received FDA accelerated approval as the first Hunter syndrome therapy specifically designed to treat neurologic manifestations by crossing the blood–brain barrier. The drug uses Denali’s TransportVehicle platform to fuse the IDS enzyme with a transferrin‑receptor...
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Oct 14, 2025
FDA Approves Boehringer’s JASCAYD as First New IPF Therapy in Over a Decade; Denali Therapeutics’ Tividenofusp Alfa BLA Review for Hunter Syndrome Extended by FDA; Bicara’s Ficerafusp Alfa Earns FDA Breakthrough Tag for 1L HPV-Negative Head & Neck Cancer; BeOne Medicines’ Sonrotoclax Granted FDA Breakthrough Designation for Cancer Treatment; FDA Clears LIBTAYO for Adjuvant Use in Cutaneous Squamous Cell Carcinoma
FDA Approves Boehringer’s JASCAYD as First New IPF Therapy in Over a Decade Boehringer Ingelheim’s JASCAYD (nerandomilast) has received FDA approval as an oral treatment for adults with idiopathic pulmonary fibrosis (IPF), marking the first new therapy for the condition in over a decade. JASCAYD is the first and...
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Aug 19, 2025
Precigen’s PAPZIMEOS Wins Full FDA Approval for Recurrent Respiratory Papillomatosis; Tonix’s Tonmya Becomes First FDA-Approved Fibromyalgia Treatment in Over 15 Years; FDA Extends BLA Review for REGENXBIO’s RGX-121 in MPS II Patients; SystImmune’s Izalontamab Brengitecan (EGFRxHER3 ADC) Gets FDA Breakthrough Designation for EGFR-Mutated NSCLC; Soligenix Granted FDA Orphan Drug Status to SGX945 for Behçet’s Disease Therapy Following Phase II Success
Precigen’s PAPZIMEOS Wins Full FDA Approval for Recurrent Respiratory Papillomatosis Precigen, Inc. announced that the FDA has granted full approval to PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). The decision makes PAPZIMEOS the first and on...
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May 22, 2020
Hunter Syndrome Market experiences a positive push as the pharma companies spot the untapped opportunities
The Hunter Syndrome Market is expected to grow with CAGR of 7.5% in the coming years in the 7MM. Occurring in approximately 1 in 100,000 to 1 in 170,000 male births, Hunter Syndrome, is an X-linked disorder, of carbohydrate metabolism that occurs almost exclusively in males, with females as the carriers of the d...
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