Regeneron Pharmaceuticals shared positive results from the company’s Phase III trial of a candidate drug for children with homozygous familial hypercholesterolemia (HoFH), a rare, life-threatening condition characterized by elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD.

The U.S. Food and Drug Administration approved Agios Pharmaceuticals Inc.’s oral PK activator Pyrukynd (mitapivat) as the first approved disease-modifying therapy for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. 

Japan’s Takeda Pharmaceutical announced deals with two biotech companies, Seattle-based Immusoft and San Diego’s Poseida Therapeutics.

The U.S. Food and Drug Administration accepted for filing the New Drug Application submitted by Acer Therapeutics Inc. and the company’s collaboration partner Relief Therapeutics Holding SA for ACER-001 (sodium phenylbutyrate) for the treatment of patients with Urea Cycle Disorders.

Albireo Pharma, a spinout of AstraZeneca, won regulatory approval for the first drug aimed at pruritus in patients with all subtypes of progressive familial intrahepatic cholestasis (PFIC).

Pivoting from conflicting and controversial press over the company’s approved Alzheimer’s disease treatment, Biogen announced that its gene therapy drug timrepigene emparvovec failed to show a clinically meaningful benefit for a rare inherited eye disease in a Phase III trial.

Biogen reported that the company’s gene therapy, cotoretigene toliparvovec, failed to hit the primary endpoint of the Phase II/III XIRIUS trial for X-linked retinitis pigmentosa (XLRP).

AstraZeneca spinout Albireo Pharma is moving closer to securing regulatory approval for the company’s treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).

The FDA approved San Diego-based Retrophin Inc.’s 100 mg and 300 mg tablets of Thiola EC (tiopronin) to treat cystinuria.

Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.