FDA updates for the week of July 18, 2022

The FDA approves Xalkori for rare tumors, as well as Opzelura for skin conditions. The agency has accepted additional indication applications for the biosimilar Hyrimoz and pegcetacoplan for advanced macular degeneration. In addition, Acadia submits an NDA for trofinetide.

The FDA approves Xalkori for a rare type of tumor.

The FDA has approved Pfizer’s Xalkori (crizotinib) for an additional indication: to treat adult and pediatric patients 1 year of age and older with unresectable, recurrent or refractories. Myofibroblastic tumors, clusters of immune cells, are rare.

The approval was based on the safety and efficacy of two trials that included 14 pediatric patients from the ADVL0912 trial and seven adult patients from the A8081013 trial. For pediatric patients, 12 of 14 patients showed an objective response. For adult patients, five patients presented an objective response.

Xalkori, first approved in 2011, is also approved for the treatment of ALK- or ROS1-positive non-small cell lung cancer and ALK-positive large cell lymphoma in children and young adults.

The FDA approves Opzelura for the skin condition.

The FDA has approved Incyte’s Opzelura (ruxolitinib) cream for the topical treatment of vitiligo in adult and pediatric patients 12 years of age and older. Vitiligo is a chronic autoimmune disease characterized by depigmentation of the skin. Opzelura is the first FDA-approved treatment for this condition and is the only topical formulation of a Janus kinase (JAK) inhibitor approved in the United States. Overactivity of the JAK signaling pathway is thought to be the root of the inflammation involved in vitiligo.

The approval comes after the FDA extended Opzelura’s review to consider additional data from clinical studies. The original Prescription Drug User Fee Act (PDUFA) date was April 18, 2022. In September 2021, Opzelura was approved by the FDA for the short-term topical treatment of mild to moderate atopic dermatitis in patients 12 years of age. and more. . It is the first topical JAK inhibitor approved for atopic dermatitis.

FDA accepts sBLA for biosimilar Hyrimoz.

The FDA has agreed to review Novartis’ Supplemental Biologics License Application (sBLA) for a high-strength formulation of Sandoz’s biosimilar Hyrimoz (adalimumab-adaz). Application includes indications for AbbVie’s reference drug Humira (adalimumab), including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis and plaque psoriasis .

If approved, the high-strength 100 mg/mL Hyrimoz would offer a reduced injection volume and potentially decrease the number of injections needed for patients who require an 80 mg dose. Hyrimoz HCF will have the same auto-injector as Hyrimoz 50 mg/mL.

FDA accepts NDA for pegcetacoplan for advanced macular degeneration.

The FDA has accepted and granted Priority Review Designation for New Drug Application (NDA) for pegcetacoplan. Developed by Apellis Pharmaceuticals, pegcetacoplan is an investigational, targeted C3 therapy for the treatment of patients with geographic atrophy secondary to age-related macular degeneration (AMD). The therapy is designed to be injected into the back of the eye. The FDA has assigned a target action date for the Prescription Drug User Fee Act (PDUFA) of November 26, 2022.

Geographic atrophy is an advanced form of age-related macular degeneration and a major cause of blindness that affects more than 5 million people worldwide, including 1 million people in the United States. There is currently no treatment for geographic AMD.

Acadia submits an NDA for trofinetide.

Acadia Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. Rett syndrome is a rare genetic neurological disorder, mostly seen in girls, and is caused by X chromosome mutations in a gene called MECP2. It causes a progressive loss of motor skills and the ability to communicate.

If approved, it would be the first therapy to treat patients with Rett syndrome, a rare genetic neurological condition in girls. Treatment currently focuses on symptom management.

Trofinetide is a novel synthetic analogue of the amino-terminal tripeptide of insulin-like growth factor-1 (IGF-1) designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function. It has been shown to inhibit the production of inflammatory cytokines, inhibit overactivation of microglia and astrocytes, and increase the amount of available IGF-1 that can bind to IGF-1 receptors.

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