Blueprint Medicines Corp. scored a broader label from the U.S. FDA for Ayvakit (avapritinib), which became the first approved therapy to treat adults with indolent systemic mastocytosis (ISM).
Once weekly vs. once daily injections plus increased potency vs. Gattex (linaclotide) inspired Ironwood Pharmaceuticals Inc. in its $1 billion takeover of Vectivbio Holding AG, which brings aboard the phase III-stage synthetic glucagon-like peptide-2 analog apraglutide, potentially a best-in-class agent for short bowel syndrome with intestinal failure.
Trouble presaged by U.S. FDA concerns over potential drug-induced liver injury (DILI) caused by obeticholic acid (OCA) 25 mg came to pass during the Gastrointestinal Drugs Advisory Committee meeting May 19 on Intercept Pharmaceuticals Inc.’s accelerated approval effort with the compound.
Word from Immix Biopharma Inc. of updated data due with NXC-201 brought to the forefront an ongoing push by drug developers to come up with a treatment for AL amyloidosis. Immix has the only CAR T therapy in the works for the disease, and the principal investigator in the Nexicart-1 phase Ib/IIa effort is slated to speak May 19 during the annual meeting of the American Society of Gene & Cell Therapy.
With PTC Therapeutics Inc.’s positive results in phase III with sepiapterin for pediatric and adult patients with phenylketonuria (PKU), attention turned to the would-be showdown with Kuvan (sapropterin dihydrochloride), the drug from Biomarin Pharmaceuticals Inc. that was approved in December 2017.
Sarepta Therapeutics Inc.’s balloting March 12 from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (OTAT) in favor of gene transfer therapy SRP-9001 (delandistrogene moxeparvovec) in Duchenne muscular dystrophy (DMD) had Wall Street mulling the odds for others in the space.
Ray Therapeutics Inc.’s upsized and oversubscribed $100 million series A financing will support the firm’s ongoing efforts with optogenetics, an approach that deploys adeno-associated virus (AAV) gene therapy to deliver a light-sensitive, highly bioengineered protein found in nature to retinal cells.
The debate over Sarepta Therapeutics Inc.’s gene transfer therapy, SRP-9001 (delandistrogene moxeparvovec), in Duchenne muscular dystrophy (DMD) proved as thorny as expected during a closely watched meeting of the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. Panelists voted on a single question: “Do the overall considerations of benefit and risk, taking into account the existing uncertainties, support accelerated approval of SRP-9001, using as a surrogate endpoint expression of Sarepta’s microdystrophin at week 12 after administration, for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene?” Balloting turned out 8 yes, 6 no.
How grave they might be remains unknown, but regulatory questions have surfaced in briefing documents related to the soon-to-happen panel meeting on Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001.
On the heels of the marketing OK in Europe, Protalix Biotherapeutics Inc. and the Chiesi Group’s global rare diseases unit scored approval of Elfabrio (pegunigalsidase alfa-iwxj) from the U.S. FDA for adults with Fabry disease.
