A host of innovative drug modalities were approved, including the first single-chain nanobody approved in the United States, an antibiotic against Gram-negative bacteria, two drugs for sickle cell anemia and a triple-drug combo covering 90% of people with cystic fibrosis. Two RNA-based therapies also received approval: an antisense oligonucleotide against Duchenne’s muscular dystrophy and the second approved small interfering RNA drug (in this case for porphyria). Meanwhile, a cocktail of monoclonal antibodies (mAbs) against three Ebola virus glycoprotein epitopes outperformed ZMapp, the current standard for treatment. In the coming quarter, regulators will review a splice-modulating small molecule for spinal motor atrophy, as well as three gene therapies: against bladder cancer, hemophilia A and metachromatic leukodystrophy.
Drug/company | Indication | Drug information |
|---|
SGT-001/Solid Biosciences | Duchenne muscular dystrophy | 11/12/2019 FDA put a clinical hold for the second time on this adeno-associated viral vector-mediated gene therapy that differentially expresses the micro-dystrophin 5 gene via the muscle-specific promoter CK8 due to serious adverse event |
Seladelpar/CymaBay Therapeutics | Primary biliary cholangitis and non-alcoholic steatohepatitis (NASH) | 11/25/2019 FDA put a clinical hold of small molecule PPAR-δ inhibitor because of atypical histological findings |
RVT-802/Enzyvant | Primary immunodeficiencies | 12/5/2019 FDA issued a complete response letter for this allogeneic thymic tissue due to issues at the manufacturing site |
Palovarotene/Ipsen | Fibrodysplasia ossificans progressive | 12/6/2019 Partial clinical hold of retinoic acid receptor agonist due to early plate closure in children |
Cellspan esophageal implant/Biostage | Esophageal cancer | 11/27/2019 FDA issued a partial clinical hold on these adipose-cell-derived stem cells seeded on proprietary scaffold until questions on the IND application are addressed |
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