Regulation

Novartis gets FDA RMAT designation for Kymriah in follicular lymphoma

Novartis has secured regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its Kymriah (tisagenlecleucel) for an investigational new indication to treat patients with relapsed or refractory (r/r) follicular lymphoma (FL).

Nexus Pharmaceuticals announces approval of Emerphed injection

Nexus Pharmaceuticals announced that has received U.S. Food and Drug Administration (FDA) approval for its patent-pending New Drug Application (NDA) Emerphed, the first and only premixed ephedrine in a ready-to-use 50 mg/10 mL vial. With no need for compounding, diluting, or mixing, Emerphed saves valuable staff time while also reducing the potential for preparation error and waste.

Boehringer Ingelheim gets EC approval for nintedanib to treat SSc-ILD

Boehringer Ingelheim has secured approval from the European Commission (EC) for its nintedanib to treat systemic sclerosis-associated interstitial lung disease (SSc-ILD) in adult patients.

FDA approves Incyte’s Pemazyre as first targeted treatment for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma

Incyte announced that the U.S. Food and Drug Administration (FDA) has approved Pemazyre™ (pemigatinib), a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

Seattle Genetics gets FDA nod for breast cancer drug Tukysa

Seattle Genetics has secured approval from the US Food and Drug Administration (FDA) for its Tukysa (tucatinib) to treat people with advanced unresectable or metastatic HER2-positive breast cancer.

FORMA Therapeutics receives FDA orphan drug designation for FT-4202 to treat sickle cell disease

FORMA Therapeutics, a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for FORMA’s lead investigational agent, FT-4202, currently in clinical development as a potentially disease-modifying treatment for sickle cell disease (SCD).

Alnylam receives fast track designation for Vutrisiran to treat polyneuropathy of hATTR amyloidosis

Alnylam Pharmaceuticals, the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to vutrisiran, an investigational therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Ascletis receives IND approval for its HIV drug ASC09F

Ascletis Pharma announces that it received IND approval from National Medical Products Administration (NMPA) for its human immunodeficiency virus (HIV) protease inhibitor ASC09F (ASC09/Ritonavir Fixed-Dose Combination).

AstraZeneca, Merck secure FDA nod for Koselugo to treat paediatric patients with NF1 genetic disorder

AstraZeneca and Merck have secured approval from the US Food and Drug Administration (FDA) for Koselugo (selumetinib) to treat paediatric patients aged two years and older with neurofibromatosis type 1 (NF1) who have symptomatic and inoperable plexiform neurofibromas (PN).