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FDA approves new genetic disorder drug

Summary by Ground News

The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of noncancerous tumors on nerves throughout the body.

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Reuters

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US FDA approves SpringWorks' genetic disorder drug

The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare genetic disorder, which causes tumors to grow in the tissue that covers nerves throughout the body.

2 months ago·United Kingdom

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The Hill

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FDA approves new genetic disorder drug

The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of noncancerous tumors on nerves throughout the body. The federal agency said on Tuesday it greenlighted SpringWorks Therapeutics’s drug Gomekli for patients dealing with neurofibromatosis type 1 (NF1) “who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.” …

2 months ago·Washington, United States

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Market Screener

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US FDA approves SpringWorks' genetic disorder drug

The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare genetic disorder, which causes tumors to grow on nerves throughout the...

2 months ago

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MedPage Today

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FDA OKs Mirdametinib for Neurofibromatosis Type 1

(MedPage Today) -- The FDA on Tuesday approved mirdametinib (Gomekli) for adults and kids ages 2 and up with neurofibromatosis type 1 (NF1). Approval of the MEK inhibitor is specifically for patients who have symptomatic plexiform neurofibromas...

2 months ago·New York, United States

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Muscular Dystrophy Association

FDA Approves New Tablet Form of Evrysdi for Treatment of Spinal Muscular Atrophy

The Muscular Dystrophy Association celebrates this new more accessible treatment option for people living with SMA. New York, Wednesday, February 12, 2025 — The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam (Evrysdi) in tablet form, marking a significant advancement in the treatment of spinal muscular atrophy (SMA), the leading genetic cause of inf…

2 months ago

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Medscape

FDA Approves Mirdametinib for Neurofibromatosis Type 1

The kinase inhibitor is now approved for adults and children aged ≥ 2 years with neurofibromatosis type 1 who have unresectable symptomatic plexiform neurofibromas.

2 months ago·United States

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