In 2021, the FDA’s most concerned action is not drug approval. Across the country and around the world, people are paying close attention to the regulatory development of Covid-19 vaccines and therapies on the road to emergency authorization. So far, of the three Covid vaccines that have received emergency authorization, Pfizer and BioNTech vaccines are the only vaccines fully approved by the FDA.
This Covid vaccine called Comirnaty is one of 60 vaccines New medicine or Biological Products Obtained FDA approval in 2021. Compared with the 61 new drugs and biological products approved by the FDA last year, the total number has dropped slightly.
As in previous years, anti-cancer drugs dominate the list of approved therapies in 2021. But there are other notable developments. In 2021, regenerative medicine received new approvals. Mallinckrodt Pharmaceuticals is approved by Stratagraft, An engineered skin product, used as a scaffold, on which the skin cells of burn patients can grow.The agency also approved a therapy for the first time Treatment of congenital athymia, A rare situation, a baby is born without a thymus, which is a key organ for the maturation of T cells. Enzyvant Therapeutics’ treatment involves implanting donor thymus tissue to rebuild immunity.
. Another difference in the approved drug list is the representation of drugs for neurological diseases.Perhaps the most compelling of these neuropharmaceuticals is Biogen’s Alzheimer’s disease drug Aduhelm received controversial FDA approval in JuneThe drug’s record in clinical trials is mixed, but regulators have chosen an accelerated approval route to bring the drug to the market and make it the first new treatment for Alzheimer’s disease in nearly 20 years.Other new neurological drugs this year include Quelbree of Supernus Pharmaceuticals, A method to treat attention deficit hyperactivity disorder; Lybalvi of Alkermes treats depression; and Qulipta, an AbbVie drug developed to prevent migraines.
Rare disease drugs are also well represented in the list of newly approved therapies. There are few treatments available for many rare diseases. But this year’s regulatory decision introduced new drug options for some of these diseases. Sarepta Therapeutics is approved by Amondys 45, The company’s third Duchenne muscular dystrophy drug. Apellis Pharmaceuticals gave the green light to Empaveli, A treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria. The drug now competes with a blockbuster drug sold by AstraZeneca’s Alexion subsidiary.Sanofi won Nexviazyme received approval this year, A method to treat Pompe disease of hereditary enzyme deficiency. The drug is Pompe’s second approved therapy, joining the ranks of Sanofi drugs. Approved in 2006.
Some rare diseases received treatment for the first time in 2021. For example, Albireo Pharma’s drug Bylvay becomes the first drug approved to treat pruritus, Or severe itching, is related to a rare liver disease with progressive familial intrahepatic cholestasis. Pruritus is related to a variety of liver diseases, and pruritus drugs to treat other liver diseases have also passed supervision this year. Cara Therapeutics is approved by Korsuva, To treat itching in patients undergoing dialysis. Alagille syndrome is a disease that affects the bile ducts of the liver and can also cause itching. Mirum Pharmaceuticals’ drug Livmarli is approved for the treatment of itching caused by the disease. These three pruritus drugs were not only the first approved drugs under their respective conditions, but also the first approved products of the companies that developed them.
Very rare diseases pose challenges because the small number of patients makes it difficult to find enough patients for well-controlled studies. But there are still ways to generate clinical data to support regulatory submissions. BridgeBio Pharma has developed its drug Nulibry for the treatment of type A molybdenum cofactor deficiency, a metabolic disorder estimated to affect less than 150 people worldwide. The natural course of the 13 patients evaluated by BridgeBio’s drug was compared with those of patients diagnosed with the disease. Based on this comparison, FDA approved Nulibry, making it the first drug approved for the treatment of this ultra-rare disease and the first BridgeBio product approved.
Among the statistics of cancer drug approvals in 2021, it is worth noting the continuing trend of targeted therapy for subgroups of patients whose diseases show specific genetic characteristics. EMD Serono drug Teppetko approved The treatment of non-small cell lung cancer (NSCLC) is characterized by genetic changes in a gene called MET. The drug is now competing with Novartis’ targeted therapy that addresses the same gene mutation.
May, the FDA approves Johnson & Johnson’s infusion therapy Rybrevant It is used to treat non-small cell lung cancer with specific mutations, called epidermal growth factor.Takeda Pharmaceutical is now using it Newly approved oral drug ExkivityCancer research is also expanding its scope to targets previously thought to be impossible to make medicines. Amgen’s new drug Lumakras solves one such goal, tracking KRAS protein mutations About lung cancer cells.
Photo courtesy of FDA
