Precision Meds Continue Gaining FDA Approvals

(Pete Linforth, Pixabay. https://pixabay.com/illustrations/dna-life-biotechnology-evolution-4068826/)

22 Feb. 2022. Food and Drug Administration approved 17 new medications in 2021 addressing genetic targets, according to data from a precision medicine advocacy group. The annual report of the Personalized Medicine Coalition in Washington, D.C. indicates both the number and percentage of new precision treatments approved by FDA in 2021 are similar but down slightly from 2020.

Precision or personalized medicines are prescribed by physicians to meet a patient’s molecular make-up, based on results of diagnostics, and the individual’s medical history. The Personalized Medicine Coalition’s annual report says FDA approved 17 new drugs and biologics meeting this definition in 2021, making up 35 percent of all new therapies last year. In 2020, FDA approved 20 new precision drugs and biologics, comprising 39 percent of all new treatments. Since 2015, precision medications account for 25 to 42 percent of all new drugs or biologics approved each year by FDA.

Seven of the 17 new precision drugs and biologics approved by FDA are cancer therapies, mainly for treating non-small cell lung cancer, but also bile duct and uterine cancer, and chronic myeloid leukemia. Each of these therapies addresses specific biomarkers, molecular indicators revealed through genetic testing. In addition, the report notes approval of two cell therapies approved by FDA last year to treat blood related cancers, large B-cell lymphoma and multiple myeloma. In both cases, patients’ T-cells are genetically altered, then re-infused as therapeutics.

New or expanded precision medicine diagnostics

FDA approved another seven new precision drugs or biologics to treat rare or inherited diseases indicated by specific biomarkers, according to the report. The approved medications treat Duchenne muscular dystrophy, Pompe disease, Von Hippel-Lindrau disease, progressive familial intrahepatic cholestasis, myasthenia gravis, heterozygous familial hypercholesterolemia, and molybdenum cofactor deficiency. Three other approved precision therapies address HIV, and severe cases of itching (pruritis) and blood-vessel inflammation (vasculitis).

The PMC report points out related FDA actions in 2021 authorizing diagnostic tests for precision therapies. The group says the agency cleared or broadened the scope of nine companion diagnostics for precision drugs. The report also highlights recognition for the Oncology Knowledge Base at Memorial Sloan Kettering Cancer Center in New York, as one of FDA’s human genetic variant databases that document evidence of links between genetic variations and diseases or conditions.

PMC president Edward Abrahams says in an organization statement the findings show “personalized medicines have accounted for more than a quarter of newly approved drugs for each of the last seven years and for more than a third of the drugs approved since 2017, not to mention the approval of new or expanded indications for a countless number of paradigm-shifting diagnostic tests,” adding that the report “leaves no question that the era of personalized medicine is upon us, presenting both opportunities and challenges for patients and health systems.”

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• Precision Cancer Meds Company Raises $43M in Early Funds
• New Biotech Creating Precision Cancer Therapies

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