• The U.S. Food and Drug Administration plays an important role in helping to address the high cost of medicines through the development of science-based policies that can improve competition and patient access to safe and effective, high-quality and lower cost generic drugs, biosimilars and interchangeable biologics.

  • U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Vyndaqel and Vyndamax have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis and their recommended doses differ.

  • The U.S. Food and Drug Administration  approved Ruzurgi (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. This is the first FDA approval of a treatment specifically for pediatric patients with LEMS. The only other treatment approved for LEMS is only approved for use in adults.

  • The U.S. Food and Drug Administration announced today the approval of Dengvaxia, the first vaccine approved for the prevention of dengue disease caused by all dengue virus serotypes (1, 2, 3 and 4) in people ages 9 through 16 who have laboratory-confirmed previous dengue infection and who live in endemic areas. Dengue is endemic in the U.S. territories of American Samoa, Guam, Puerto Rico and the U.S. Virgin Islands.

  • The U.S. Food and Drug Administration  approved a device, called Xvivo Perfusion System with Steen Solution Perfusate, that can temporarily ventilate, oxygenate, and pump (perfuse) preservation solution through lungs that were initially thought to be unacceptable for transplant. The device allows the transplant team to perform a more careful assessment of lung function to get a better sense of how the organs are working when they are perfused with a solution outside the body to better determine whether the lungs can then be viable for transplant.

  • The U.S. Food and Drug Administration granted accelerated approval to Balversa (erdafitinib), a treatment for adult patients with locally advanced or metastatic bladder cancer that has a type of susceptible genetic alteration known as FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy. Patients should be selected for therapy with Balversa using an FDA-approved companion diagnostic device.

  • U.S. Food and Drug Administration is warning the public not to use medical devices marketed to consumers that claim to help assess, diagnose or manage head injury, including concussion, traumatic brain injury (TBI) or mild TBI. In a new safety communication, the FDA warned that such tools — such as apps on a smartphone marketed to coaches or parents for use during sporting events — have not been reviewed by the FDA for safety and efficacy and could result in an incorrect diagnosis, potentially leading to a person with a serious head injury returning to their normal activities instead of getting medical care.

  • U.S. Food and Drug Administration issued a warning letter to Inova Genomics Laboratory (Inova) of Falls Church, Virginia, for illegally marketing certain genetic tests that have not been reviewed by the FDA for safety and effectiveness. The tests claim to predict patients’ responses to specific medications based on genetic variants. Selecting or changing drug treatment in response to the test results could lead to potentially serious health consequences for patients.

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