Two biologic therapeutic candidates met their primary endpoints in separate Phase III trials: AstraZeneca’s Ultomiris and Argenx’s Vyvgart.

Aeglea BioTherapeutics announced on April 11 promising Phase III results for patients diagnosed with arginase 1 deficiency (ARG1-D), a rare hereditary metabolic disorder. Aeglea said on April 12 the company had submitted its first-ever Biologics License Application (BLA) to the U.S. Food and Drug Administration for the drug.

Theravance Biopharma announced mixed results from a study evaluating the use of ampreloxetine to reduce symptomatic neurogenic orthostatis hypotension (nOH).

The U.S. Food and Drug Administration approved Sanofi’s proposed drug to address red blood cell destruction in patients living with a rare form of anemia called cold agglutinin disease (CAD). 


With two assets licensed from Takeda, Oak Hill Bio launched with a focus on developing therapeutics for patients and extremely preterm infants who are suffering from rare autoimmune diseases.

Biohaven Pharmaceutical Holding Co. Ltd. said on Sept. 27 the company’s drug candidate, verdiperstat, failed to show effectiveness in treating a rare neurological disorder called multiple system atrophy (MSA).

Investors Hand Disc Medicine $90 Million to Control Hematological Diseases Published: Sept. 2, 2021 By Heather McKenzie BioSpace   A two-year-old biopharma company tackling rare blood diseases has received a […]


AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.

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.

The U.S. Food and Drug Administration approved Pfizer Inc.’s supplemental Biologics License Application for Panzyga to treat adult patients with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP).