FDA sign, headquarters

FDA mark, headquarters

Cancer drug research is producing more targeted therapies that target specific genetic signatures. Last year saw our fair share of new targeted cancer therapies, but 2022 was also notable for the expansion of genetic insights beyond oncology. Three gene therapies have won FDA approval, and the growing list of such therapies in clinical trials suggests the trend will continue this year.

The three newly approved gene therapies are among the 42 medicines and biological products that won FDA approval in 2022. This total excludes imaging agents and diagnostic products, some of which are on FDA lists. Also excluded from the total are previously approved drugs that have received additional approvals for new indications. The number of new therapies approved in 2022 is less than 60 new products approved in 2021a year in which Covid-19 sparked an influx of research dollars that spread to the drug research sector and led to the first FDA-approved vaccines for the novel coronavirus.

The first gene therapy to win FDA approval in 2022 was Zynteglo, Bluebird Bio treatment for the rare blood disorder beta thalassemia. The biotech company followed that regulatory decision with approval for Skysona, a gene therapy for cerebral adrenoleukodystrophy, an extremely rare neurodegenerative disease. But gene therapy is expanding to conditions that, while still rare, concern patient populations measured in the tens of thousands, not the thousands. Hemophilia B is one such disease.

CSL Behring’s approval of Hemgenix made this product the first approved gene therapy for hemophilia B. The approval also set a record for pricing a therapy of any kind. CSL charges $3.5 million for its therapy, which exceeds the prices of both Bluebird Bio products.

Gene therapy offers the potential to address the genetic causes of disease with a single treatment. Thus, the potentially curative effects of gene therapy come with premium prices. However, it is worth noting that all of these products come with value-based agreements that tie the payer’s reimbursement of the therapy to certain outcomes demonstrating patient benefit. Industry observers say that as gene therapies become more widespread, such agreements will become more common. Meanwhile, more gene therapies are on the way. Regulatory decisions to watch in 2023 include a BioMarin Pharmaceutical gene therapy for hemophilia A and Bluebird gene therapy for sickle cell disease.

Moving on from gene therapy but remaining in the realm of rare diseases, 2022 saw several diseases receive their first approved treatments. Sanofi drug Enjaymo and Agios Pharmaceuticals’ Pyrukynd are the first approved therapies for anemias caused by cold agglutinin disease and pyruvate kinase deficiency, respectively. Acid sphingomyelinase deficiency, a rare disease characterized by the absence of a key enzyme, now has its first FDA-approved treatment: Sanofi’s Xenpozyme. The US approval for Xenpozyme followed by approval of this product in Europe and Japan. In other metabolic drug news, Eli Lilly wins regulatory approval for Mounjaro, a type 2 diabetes drug expected to become a hit seller.

Perhaps the most high-profile approval for 2022 was the decision for Relyvrio, Amylyx Pharmaceuticals’ treatment for amyotrophic lateral sclerosis. Two FDA advisory committee meetings were held last year on the drug — unusual for a single product. Along the way, biotechnology navigated uncertainty about whether another clinical trial is needed to support its regulatory submission.

Ultimately, the FDA concluded that it could review Relyvrio based on the results of a Phase 2 study. But in a surprise move, the agency granted the drug standard approval rather than accelerated approval. Relyvrio’s regulatory nod in September made it only the third drug approved so far for ALS.

Cancer therapies are a sure bet at the top of annual FDA-approved new product lists, and 2022 had its share of new oncology products. But compared to previous years, this past year has been relatively slow for new cancer drugs. A total of 14 new anticancer products passed regulatory scrutiny. One of the closest of these drugs was Mirati Therapeutics’ Krazati for non-small cell lung cancer. The small molecule targets a rare mutation called KRAS G12C, and approval of the Mirati drug puts it in direct competition with Amgen’s Lumakras.

Multiple myeloma is another type of cancer welcoming new products. Carvykti, a CAR-T therapy from Johnson & Johnson and Legend Biotech, is one of two multiple myeloma drug approvals for J&J this year. The agency too approved Tecvayli, J&J’s antibody drug that pursues the same goal as Carvykti. However, Tecvayli’s subcutaneously injected formulation is intended to offer patients a more convenient dosing option compared to IV cell therapies.

J&J may soon add a fifth therapy to its multiple myeloma product lineup. During the recent annual meeting of the American Society of Hematology, the pharmaceutical giant presented data supporting its application for talquetamab, which could become the first drug approved to address the new cancer target GPRC5D. This will be one of the regulatory decisions to watch for in 2023.

Photo: Getty Images, Sarah Silbiger

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