Truist Sees Positive Readthrough for Maze After Vertex Kidney Data

Vertex Pharmaceuticals announced new interim data showing that ALYFTREK, or vanzacaftor/tezacaftor/deutivacaftor, restored exocrine pancreatic function in some children with cystic fibrosis ages 2 to 5, allowing them to stop pancreatic enzyme replacement therapy. The data, presented as a late breaker poster at the North American Cystic Fibrosis Conference, came from 48 children enrolled in the PERT discontinuation substudy VX22-121-106 Cohort 2, part of an ongoing open-label extension study in that age group. After at least 48 weeks of treatment, mean fecal elastase-1 was 239.7 micrograms per gram, a mean increase of 110.2 micrograms per gram from baseline, and 48.6 percent of children reached the pancreatic sufficiency threshold of at least 200 micrograms per gram. Of the 48 children eligible for the substudy, 18, or 37.5 percent, successfully discontinued pancreatic enzyme replacement therapy for a mean duration of 8.6 weeks, and across the full enrolled cohort of 66 children, 50 percent were not on the therapy at the data cut. Carmen Bozic, Vertex's chief medical officer, said the findings represent a profound shift in understanding the benefits of treating cystic fibrosis in its earliest stages, noting that pancreatic failure had long been considered irreversible. Vertex also presented additional abstracts at the conference, held October 7 to 10 in Atlanta, on clinical and real-world evidence for CFTR modulators, including a post-hoc analysis of reduced intravenous antibiotic use with VNZ/TEZ/D-IVA in patients 12 and older and a natural history study of exocrine pancreatic function in infants under 12 months. The use of ALYFTREK in children 2 to 5 years old is investigational.

Scholar Rock has resubmitted its Marketing Authorisation Application for apitegromab to the European Medicines Agency for the treatment of children and adults with spinal muscular atrophy. The resubmitted application replaces the Catalent Indiana fill-finish facility with Scholar Rock's alternate fill-finish facility, the same U.S.-based site that supported the September 2026 FDA approval of ISEMBYLD, also known as apitegromab-mstn. The company withdrew its previous MAA in August 2026 after a lack of GMP compliance at the Catalent Indiana facility, and now anticipates a Committee for Medicinal Products for Human Use opinion in the first half of 2027. Chief Executive Officer David L. Hallal said the company is working with urgency to bring this first-ever muscle-targeted therapy to children and adults living with SMA in Europe, where more than 35,000 SMA patients globally are receiving an SMN-targeted therapy. Apitegromab is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA currently receiving a survival motor neuron 2-targeted treatment, based on the Phase 3 SAPPHIRE study.

The pheochromocytoma and paraganglioma market across the United States, EU4, the United Kingdom and Japan is projected to grow from approximately USD 300 million in 2025 to approximately USD 670 million by 2036, a CAGR of 7.3% during 2026-2036, according to a new ResearchAndMarkets.com report. The United States represented the largest regional market, generating approximately USD 200 million in 2025, and Merck's belzutifan, sold as WELIREG, is forecast to generate the highest PCPG therapy revenue in the United States by 2036. WELIREG, a selective HIF-2a inhibitor and the first oral targeted PCPG therapy, was approved in May 2025 for adult and pediatric patients aged 12 years and older with locally advanced, unresectable or metastatic PCPG, at a US list price of USD 31,162.50 per bottle of 90 tablets, or an estimated annual cost of USD 373,950. Approximately 4,900 incident PCPG cases were reported across the 7MM in 2025, including around 2,400 in the United States, and the report notes that pipeline rivals from Novartis, Jazz Pharmaceuticals and Perspective Therapeutics are advancing, including LUTATHERA, JZP3507 and VMT-a-NET.