FDA Approves Selumetinib for Rare Pediatric Neurofibromatosis

April 16, 2020 - FDA recently approved the first treatment for pediatric patients with neurofibromatosis phase 1 (NF1), a rare disease that affects children. 

NF1 is a rare, progressive condition of the nervous system caused by a mutation or flaw in a particular gene that causes tumors to grow in nerves. Koselugo (selumetinib) is a kinase inhibitor, which functions by blocking a key enzyme and stops any tumor growth.

“Everyone’s daily lives have been disrupted during the COVID-19 pandemic, and in this critical time we want patients to know that the FDA remains committed to making patients with rare tumors and life threatening diseases, and their unique needs, a top priority. We continue to expedite product development for these patients,” Richard Pazdur, MD, Director of FDA’s Oncology Center of Excellence and Acting Director of the Office of Oncologic Diseases in FDA’s Center for Drug Evaluation and Research, said in the announcement.

NF1 is so rare that it occurs in one out of ever 3,000 infants. Individuals affected by the condition will notice changes in skin coloring, neurologic and skeletal impairments, and risk for development of benign and malignant tumors. 

Koselugo is approved specifically for patients with tumors involving the nerve sheaths, or inoperable plexiform neurofibromas (PN). Between 30 percent and 50 percent of patients born with NF1 develop one or more PNs. The tumors can grow on the face, extremities, and areas around the spine and deep in the body where they may affect organs. 

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“We are committed to regulatory flexibility and providing extensive guidance to industry in an effort to bring drugs forward that fulfill unmet medical needs. Koselugo represents this commitment,” Pazdur noted. “For the first time, pediatric patients now have an FDA-approved drug to treat plexiform neurofibroma, a rare tumor associated with NF1.”

FDA approved Koselugo based on outcomes of a clinical trial conducted by the National Cancer Institute of pediatric patients who had NF1 and inoperable PN. The clinical trial showed that out of 50 patients, the overall response rate was 66 percent and all patients had a partial response. Of these patients, 82 percent had a response lasting 12 months or longer. 

FDA granted the application priority preview and breakthrough therapy designation. Koselugo also received orphan drug designation and rare pediatric disease designation for the treatment of pediatric NF1.

FDA Approves First Generic of a Commonly Used Albuterol Inhaler to Treat and Prevent Bronchospasm

FDA also recently  the first generic of Proventil HFA (albuterol sulfate) Metered Dose Inhaler, 90mcg/ Inhalation, to treat bronchospasm, a chronic disease, which causes sudden constriction of the muscles in the walls of the bronchioles.

The dosage is specifically given to individuals four years of age or older who have reversible obstructive airway disease, and to preventexercise-induced bronchospasm. 

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“The FDA recognizes the increased demand for albuterol products during the novel coronavirus pandemic,” said FDA Commissioner Stephen M. Hahn, MD. “We remain deeply committed to facilitating access to medical products to help address critical needs of the American public.”

Bronchospasms occur when the muscles surrounding the airways tighten, causing them to squeeze the airways and make them smaller, according to the National Heart, Lung, and Blood Institute. Exercise may bring on the symptoms in individuals who have asthma. 

FDA noted that under the Generic Drug User Fee Amendments (GDUFA), individual companies can meet with the agency regarding the development of generic drug products. 

Recently, FDA issued a revised draft product-specific guidance for proposed generic albuterol sulfate metered dose inhalers, including drug products referencing Proventil HFA. The draft ensures quality generic drug products are as safe and effective as their brand name counterparts.

FDA Authorizes Blood Purification Device to Treat COVID-19

Last week, FDA administered an emergency use authorization (EUA) for a blood purification system to treat patients 18 years of age or older with COVID-19 admitted to the intensive care unit (ICU) with respiratory failure. 

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FDA issued the EUA for Terumo BCT and Marker Therapeutics AG for their Spectra Optia Apheresis System and Depuro D2000 Adsorption Cartridge devices.

The product reduces the amount of cytokines and other inflammatory mediators such as small active proteins in the bloodstream that control a cell’s immune response by filtering the blood and returning the filtered blood to the patient, the press release stated.

The proteins removed by the product are elevated during infections and may be associated with “cytokine storm,” which occurs in some COVID-19 patients. This leads to severe inflammation, rapidly progressive shock, respiratory failure, organ failure, and death. 

“We continue to work across all sectors to expedite the development of numerous innovative potential preventive and treatment approaches by both facilitating emergency access for patients, to the extent we can, and supporting the evaluation of potential therapies,” FDA Commissioner Stephen M. Hahn, MD, said in the press release.

“With today’s authorization of a blood purification device, we are expediting the availability of a treatment option for patients in the ICU to help reduce the severity of the disease. Our staff will continue our around the clock review of all medical products to expedite the availability of treatments to help fight this devastating disease.”

FDA Issues Emergency Use Authorization to Decontaminate Millions of N95 Respirators

Most recently, FDA issued a EUA that may decontaminate nearly four million N95 or N95-equivalent respirators per day in the US for frontline healthcare workers, according to a recent press release.

The EUA was granted to Advanced Sterilization Products (ASP) for the STERRAD Sterilization Cycles, which uses vaporized hydrogen peroxide gas plasma sterilization. There are currently about 9,930 systems in nearly 6,300 hospitals across the US.

The STERRAD 100S Cycle, STERRAD NX Standard Cycle, and STERRAD 100NX Express Cycle vary in reprocessing times from 55 minutes to 28 minutes to 24 minutes. The systems can reprocess about 480 respirators a day. 

“Our nation’s health care workers are among the many heroes of this pandemic and we need to do everything we can to increase the availability of the critical medical devices they need, like N95 respirators,” FDA Commissioner Stephen M. Hahn, MD, said in the press release.

“FDA staff continue to work around the clock, across government and with the private sector to find solutions. This authorization will help provide access to millions of respirators so our health care workers on the front lines can be better protected and provide the best care to patients with COVID-19.”