New Drug Approvals

The FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 48 novel drugs in 2019, however, it did not reach of 2018 record with a total of 59 approvals. Although this approval count still comes as the third biggest approval in the past 25 years.

42% of the novel drugs approved are classified as first in class and usually offers different mechanisms of action from those of existing therapies. One of these drugs is Balversa™ (erdafitinib) from the Janssen Pharmaceuticals, is a pan-fibroblast growth factor receptor indicated as a treatment for adult patients with locally advanced or metastatic bladder cancer. Another drug within this classification is Adakveo™ Injection (crizanlizumab-tmca) from Novartis Pharmaceuticals Corporation, is a P-selectin inhibitor indicated for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease.

In 2019, 21 of CDER’s 48 novel drug approvals were approved to treat rare diseases. Patients with rare diseases often have few or no drugs available to treat their conditions and this represents a breakthrough in the pharmaceutical area. One of these drugs is Turalio™ (pexidartinib) capsules. Turalio is a small molecule tyrosine kinase inhibitor that helps decrease tumor growth. Is indicated for adult patients with tenosynovial giant cell tumor, a rare condition involving non-cancerous tumors around the joint areas. Another drug is Givlaari™ (givosiran) injection for subcutaneous use from Alnylam Pharmaceuticals. Givlaari™ is an aminolevulinate synthase 1-directed siRNA indicated for the treatment of adults with acute hepatic porphyria, characterized by the occurrence of neuro-visceral attacks with or without cutaneous manifestations.

Regulatory processes are different between FDA and regulatory agencies of the rest of countries, 33 of the total of novel drugs approved in 2019 were approved in USA before receiving approval in other country.

Clinical Researchers are working to offer patients new and better treatments for different diseases and for all this participating in a clinical study is an excellent opportunity to help us.  This new year we are going to open more protocols and if you would like more information as a patient or sponsor, feel free to contact us.