The U.S. Food and Drug Administration (FDA) approved golodirsen on December 12, 2019, for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Johnson & Johnson submits application to U.S. FDA seeking approval of TREMFYA® (guselkumab) for the treatment of moderately to severely active Crohn’s disease.

The U.S. Food and Drug Administration (FDA) approved voxelotor on November 25, 2019, for the treatment of sickle cell disease in adults and children aged 12 years and older.

Neurotech Pharmaceuticals, Inc. Receives Priority Review of Biologics License Application (BLA) for NT-501 (revakinagene taroretcel) as a Treatment for Macular Telangiectasia Type 2 (MacTel).

The U.S. Food and Drug Administration (FDA) approved cenobamate on November 21, 2019, for the treatment of partial-onset seizures in adults.

Tubulis Doses First Patient in Phase I/IIa Trial Investigating ADC Candidate TUB-040 in Ovarian Cancer and Lung Adenocarcinoma.

The U.S. Food and Drug Administration (FDA) approved Givosiran on November 20, 2019, for the treatment of acute hepatic porphyria (AHP) in adults.

InnoCare Announces the Acceptance of Biologics License Application for Tafasitamab in Combination with Lenalidomide for the treatment of Relapsed or Refractory Diffuse Large B-Cell Lymphoma in Adult Patients in China.

The U.S. Food and Drug Administration (FDA) approved Crizanlizumab on November 15, 2019.

Silence Therapeutics Announces Positive Topline 48-Week Data from Phase 2 Study of Zerlasiran in Patients with Elevated Lipoprotein(a).

The U.S. Food and Drug Administration (FDA) approved Zanubrutinib on November 14, 2019.

Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above.