FDA Grants Accelerated Approval for First Barth Syndrome Treatment

20 settembre 2025 Riportato dall'IA

The U.S. Food and Drug Administration has granted accelerated approval to Forzinity, the first treatment for Barth syndrome, a rare X-linked genetic disorder primarily affecting males. Developed by Stealth Biotherapeutics, the therapy targets mitochondrial dysfunction underlying the condition, offering new hope for patients with this life-threatening disease. This milestone follows years of advocacy and highlights progress in addressing ultra-rare disorders.

Senate confirms Robert Califf as FDA commissioner

02 ottobre 2025 Riportato dall'IA

The U.S. Senate has confirmed Robert Califf to lead the Food and Drug Administration amid ongoing public health challenges. Califf, a cardiologist with prior FDA experience, was approved in a bipartisan vote. This move aims to stabilize the agency during debates over drug approvals and regulations.

FDA Approves Injectable Form of Merck's Keytruda

The U.S. Food and Drug Administration has granted approval for an injectable version of Merck's blockbuster cancer drug Keytruda, marking a significant advancement in treatment options for patients with certain types of cancer. This subcutaneous formulation promises greater convenience over the traditional intravenous method, potentially improving patient adherence and reducing healthcare burdens. The decision, announced on September 19, 2025, underscores ongoing innovations in oncology amid rising demands for more accessible therapies.

Federal judge blocks expanded access to abortion pill mifepristone

A federal judge in Texas has issued a ruling limiting the FDA's approval of mifepristone, the primary drug used in medication abortions. The decision revives a long-standing lawsuit challenging the drug's safety and regulatory process. This comes amid ongoing legal battles over abortion access following the Supreme Court's overturning of Roe v. Wade.