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FDA Updates for Week of July 18, 2022


FDA approves Xalkori for rare tumor, as well as Opzelura for skin conditions. The agency has accepted applications for a supplemental indication for the biosimilar Hyrimoz and pegcetacoplan for advanced macular degeneration. Additionally, Acadia submits NDA for trofinetide.

FDA approves Xalkori for 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 refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT). Myofibroblastic tumors, masses of immune cells, are rare.

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

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

FDA approves Opzelura for 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 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. Over-activity of the JAK signaling pathway is believed to drive inflammation involved in vitiligo.

The approval comes after the FDA had extended the review of Opzelura 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 topical short-term treatment of mild-to-moderate atopic dermatitis in patients 12 years of age and older. This is the first topic JAK-inhibitor approved for atopic dermatitis.

FDA accepts sBLA for the biosimilar Hyrimoz.

The FDA has accepted for review Novartis’ supplemental biologics license application (sBLA) for a high-concentration formulation of Sandoz’ biosimilar Hyrimoz (adalimumab-adaz). The application includes the indications of the reference medicine AbbVie’s Humira (adalimumab), including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis.

If approved, the high-concentration Hyrimoz 100 mg/mL would offer reduced injection volume and potentially decrease the number of injections required for patients who need 80 mg dosing. 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 the 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 Prescription Drug User Fee Act (PDUFA) target action date is Nov. 26, 2022.

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

Acadia submits 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 rare genetic neurological disorder mostly seen in girls, and is caused by mutations on the X chromosome on a gene called MECP2. It causes progressive loss of motor skills and the ability to communicate.

If approved, this would be the first therapy to treat patients with Rett syndrome, a rare genetic neurological disorder in girls. Treatment is currently focused on managing symptoms.

Trofinetide is a novel synthetic analog 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 the overactivation of microglia and astrocytes, and increase the amount of available IGF-1 that can bind to IGF-1 receptors.

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