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."
The drug was approved after a multicenter, single-arm trial of 114 patients — 58 adults — with “symptomatic, inoperable NF1-associated PN causing significant morbidity,” according to the FDA.
Gomekli showcased at least a 20 percent reduction in the volume of the tumor in more than 50 percent of pediatric patients and 41 percent of adult patients, Reuters noted.
“The NF1-PN patient community has a great need for more treatment options. With today’s approval, we are honored to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief,” SpringWorks CEO Saqib Islam said in a statement on Tuesday.
“We are grateful to each clinical trial participant, their families, the investigators, and the patient advocacy groups involved in the journey towards making GOMEKLI available in the U.S.,” Islam added. “I am proud that we are delivering on our commitment to patients with devastating diseases with our company’s second FDA approval in less than 18 months.”
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