Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-A501.
Vertex Pharmaceuticals (Europe) has secured approval from the European Commission (EC) for Orkambi (lumacaftor/ivacaftor) to treat of children with cystic fibrosis (CF) aged two to five years old who have two copies of the F508del mutation.
Zymeworks, a clinical-stage biopharmaceutical company developing multifunctional therapeutics, reported the achievement of a new development milestone in its collaboration with Eli Lilly and Company (“Lilly”).
The Janssen Pharmaceutical Companies of Johnson & Johnson has secured marketing approval from the European Commission (EC) for Erleada (apalutamide) to treat non-metastatic castration-resistant prostate cancer (nmCRPC).
Silence Therapeutics’ lead candidate SLN124 has secured orphan drug designation in the European Union for the treatment of β-Thalassemia.
Exelixis announced that the U.S. Food and Drug Administration (FDA) approved CABOMETYX (cabozantinib) tablets for patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.
Pfizer’s tafamidis meglumine form (20mg capsule) has secured priority review from the US Food and Drug Administration (FDA) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).
UK-based specialty pharma company Diurnal Group said that the US Patent and Trademark Office (USPTO) has granted a second US patent for Chronocort (modified release hydrocortisone).
Ocular Therapeutix is seeking approval from the US Food and Drug Administration (FDA) for Dextenza (dexamethasone ophthalmic insert) to expand the current indication to include the treatment of ocular inflammation following ophthalmic surgery.
Novartis has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for crizanlizumab (SEG101) to prevent vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD).
Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.