United States: The Food and Drug Administration approved a new medication to treat patients manifesting noncancerous tumors across entire body nerves.
Health authorities under the FDA authorized Gomekli as a treatment option for NF1 patients presenting symptomatic plexiform neurofibromas which doctors cannot remove completely, as reported by The Hills.
Clinical Trials and Approval Process
The pharmaceutical company obtained FDA approval for the drug by conducting clinical trials involving 114 subjects and specifically focusing on 58 adult patients with “symptomatic, inoperable NF1-associated PN causing significant morbidity.”
Gomekli demonstrated a minimum tumor volume reduction of 20 percent when evaluated in more than 50 percent of pediatric patients and 41 percent of adult patients according to data reported by Reuters.
Significance for the NF1 Community
“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.
Commitment to Advancing Patient Care
“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.”