Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections.
Ultragenyx Pharmaceutical Inc. and Mereo Biopharma Group plc, along with investors, had hoped for an early halt to the phase III study of setrusumab in treating brittle bones. They didn’t get it, as the clinical trial’s data monitoring committee wants the study to roll on through its final analysis.
Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage disease type 1a (GSD1a) after the phase III study hit its primary endpoint and two key secondary endpoints.
Wall Street may not have responded as positively as Ultragenyx Pharmaceutical Inc. would have liked after the firm unveiled new data from the phase I/II study with GTX-102 for the treatment of Angelman syndrome (AS). Patients in expansion cohorts A & B treated with a set dose and regimen of the intrathecally delivered antisense oligonucleotide (ASO) showed rapid and clinically meaningful improvement across multiple domains.
At first glance, the number of drugs that received accelerated approval from the U.S. FDA’s Center for Drug Evaluation and Research (CDER) in 2023 was nothing to write home about. Yes, CDER granted nine accelerated approvals last year, up from six in 2022. But the proportion of novel drugs with accelerated approval was 16% both years. And when compared with the 12 drugs in 2020 and the 14 that received accelerated approval in 2021, last year’s crop was a little lackluster. However, a deeper look at the 2023 class of accelerated approvals shows a historic milestone. For the first time since the path was created in 1992, the number of novel biologics getting accelerated approval at CDER outpaced the number of small-molecule drugs.
A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
New interim data from Ultragenyx Pharmaceutical Inc. and Genetx Biotherapeutics LLC’s phase I/II study of GTX-102 in Angelman’s syndrome didn’t do much to bolster investor confidence, as Ultragenyx shares sagged considerably July 19. The Novato, Calif.-based company’s stock (NADAQ:RARE) closed 13% downward at $52.89 per share. That is nearly half the price shares fetched in late August. At $102.40 per share, that was the stock’s highest value in the past 12 months.
Mereo Biopharma plc is heading into phase III with a bone-building drug to treat the rare condition osteogenesis imperfecta – and CEO Denise Scots-Knight reckons the company has the financial backing to get setrusumab marketed thanks to a partnership with Ultragenyx Pharmaceutical Inc.
Immunogen Inc. leads a busy earnings season with numbers that pushed the stock (NASDAQ:IMGN) 30.3% higher on Feb. 12. The antibody-drug conjugates developer posted revenues of $132.3 million for 2020 compared to its $82.3 million in revenues for 2019, a 62.2% increase.
The latest global regulatory news, changes and updates affecting biopharma, including: CBO model touts savings of H.R. 3; FDA to ODAC: Are data ripe for 2nd TNBC Keytruda approval?; Fifth Circuit reiterates no liability remedy for generics; Consultant faces insider trading charges.
