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Ascendis Pharma of Denmark got slapped with a Complete Response Letter (CRL) in which the FDA voiced concerns about the variability in the delivered dose of the company’s investigational treatment for adults with hypoparathyroidism. Read More
The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 11 to 1 with one abstention to recommend that the FDA restrict approval for the combination of AstraZeneca’s blockbuster cancer drug Lynparza (olaparib) and Zytiga (abiraterone) to just metastatic castration-resistant prostate cancer (mCRPC) patients whose tumors have a breast cancer gene (BRCA) mutation. Read More
Takeda has another shot on goal for a self-administered subcutaneous form of its blockbuster Entyvio (vedolizumab) as maintenance treatment for patients with ulcerative colitis who have completed induction therapy with the antibody’s intravenous form. Read More
Biogen has received an Accelerated Approval for Qalsody (tofersen) for the treatment of an extremely rare genetic form of amyotrophic lateral sclerosis (ALS) despite less than stellar trial results. Read More
The FDA has placed a partial clinical hold on Foghorn Therapeutics’ phase 1 dose-escalation study of FHD-609 after a patient with synovial sarcoma developed a serious heart arrythmia while taking the second-highest dose. Read More
Bluebird Bio is pulling out all the regulatory stops for its investigational sickle cell disease (SCD) gene therapy lovotibeglogene autotemcel (lovo-cel), submitting a BLA with a request for priority review in addition to the four expedited approval designations the FDA had already granted the drug. Read More
Genentech has won full approval for its first-in-class combination therapy aimed at providing curative treatment for patients with previously untreated diffuse large B-cell lymphoma (DLBCL), the most common form of non-Hodgkin lymphoma in the U.S. Read More
Before the passage of the Orphan Drug Act (ODA) in 1983, the logistical challenges and financial downsides of developing a drug for a very small population affected by a rare disease meant few drugs on the horizon. Read More