FDA Updates: Approvals in Rare Disorders; Expanded Approval for Flu Treatment


FDA approves several drugs to treat rare conditions in both cancer and metabolic diseases.

Approval in neuroblastoma. FDA on Wednesday approved Y-mAbs Therapeutics’ Danyelza (naxitamab-gqgk) for the treatment of neuroblastoma, to be used in combination with granulocyte-macrophage colony-stimulating factor. Approval is for pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. The monoclonal antibody targets the ganglioside GD2, which is expressed in various neuroectoderm-derived tumors and sarcomas. The drug is given to patients 3 times a week on an outpatient basis. Danyelza was developed at Memorial Sloan Kettering and licensed to Y-mAbs; the therapy has previously received Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations. Approval was granted under the accelerated approval process.

Therapy to manage weight in rare conditions. FDA on Friday approved Imcivree (setmelanotide) to help patients with obesity manage weight loss and ongoing weight management if they have 1 of 3 genetic conditions: pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, and leptin receptor (LEPR) deficiency, when these conditions are confirmed by genetic testing showing variants in POMC, PCSK1, or LEPR genes that cause disease or are of uncertain significance. Imcivree is the first FDA-approved treatment for these genetic conditions; these conditions are quite rare, with only around 150 reported in the medical literature for all 3 combined. Imcivree is approved for patients at least 6 years of age and is not approved for obesity due to suspected deficiency with variants classified as benign, or for other forms of obesity. Common side effects include injection site reaction, skin hyperpigmentation, and gastrointestinal effects. FDA had previously granted orphan disease designation, Breakthrough Therapy designation, and Priority Review for this drug application. Rhythm Pharmaceutical, Inc., received approval for the therapy.

First Drug to Treat Rare Metabolic Disorder. On Monday, FDA approved Oxlumo, (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1). According to a statement from FDA, approval for this rare genetic disorder comes after collaboration between experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative. Patients with PH1 produce too much oxalate, a substance that is consumed in food but also produced in the body; oxalate can combine with calcium to cause kidney stones and progressive kidney damage, which can lead to the need for dialysis. The condition affects 1 to 3 people per million in North America and Europe. Oxlumo, made by Alnylam Pharmaceuticals, Inc., decreases oxalate production. Common side effects include injection site reaction and abdominal pain.

Xofluza Approval Expanded. Also Monday, FDA expanded approval for the influenza medication Xofluza (baloxavir marboxil) to include prevention of influenza for patients 12 years of age and older after contact with an individual who has the flu. Xofluza, was previously available only as a tablet but is now also available as granules for mixing in water. Xofluza was first approved in 2018 for treating uncomplicated flu in patients 12 years of age and older who have been symptomatic for less than 48 hours. FDA granted the expanded approval to Genentech.


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