Alnylam Pharmaceuticals presented complete results from the company’s ILLUMINATE-B Phase III study in children under 6 years with primary hyperoxaluria type 1 (PH1), a rare disorder affecting the kidneys.
An experimental Amgen Inc. drug that targets a specific genetic mutation shrank tumors in 32% of advanced lung cancer patients and 7% of those with colon cancer, according to data from an early-stage trial.
Researchers at the The Scripps Research Institute found that the strains spreading so quickly in Europe and the U.S. have a mutated S “spike” protein that makes it about 10 times more infectious than the strain that originally was identified in Asia.
The U.S. Food and Drug Administration approved Clovis Oncology Inc.’s drug Rubraca for the treatment of adult patients with metastatic castration-resistant prostate cancer, whose tumors have a genetic mutation.
The first treatment for adult patients with metastatic non-small cell lung cancer (NSCLC) who also have a specific genetic mutation was approved by the U.S. Food and Drug Administration.
Germany’s drug assessment body said data provided by Bayer on the precision cancer drug Vitrakvi did not provide clear enough evidence of benefits, in a setback to the drugmaker in the company’s home market.
Merck & Co. Inc. is buying ArQule Inc. for $2.7 billion, seeking to tap into the drug developer’s experimental blood cancer therapy that targets genetic mutations.
Novartis AG won U.S. approval for the company’s experimental sickle cell disease drug Adakveo.
Vertex Pharmaceuticals Inc. priced the company’s FDA-approved Trikafta, a three-drug combination for cystic fibrosis, at $311,503 per year.
The U.S. Food and Drug Administration approved Clinuvel Pharmaceuticals Ltd.’s treatment for a rare inherited disorder that causes skin damage from exposure to light, sending the Australian drugmaker’s shares to a record high.