Top FDA News for Week of Sept. 20, 2021


Pfizer receives an EUA for its COVID-19 booster, a biosimilar of Lucentis to treat macular degeneration is approved, Incyte receives two approvals for JAK inhibitors, one for atopic dermatitis and another for GVHD, and more FDA news.

FDA issues EUA for booster of Pfizer’s COVID-19 vaccine.

The FDA has updated the emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 vaccine to allow for use of a single booster dose for: people 65 years of age and older; people through 64 years of age at high risk of severe COVID-19; and people 18 through 64 years of age at risk for occupational exposure to COVID-19.

The FDA authorized the booster to be given at least six months after completion of the primary vaccination.

FDA approves first biosimilar to treat macular degeneration.

The FDA has approved Biogen and Samsung Bioepis’ Byooviz (ranibizumab-nuna) as the first biosimilar to Genentech’s Lucentis (ranibizumab) for the treatment of several eye diseases and conditions, including neovascular (wet) age-related macular degeneration, a leading cause of vision loss and blindness for Americans over the age of 65. Byooviz is also approved to treat macular edema following retinal vein occlusion (blockage of veins in the retina) and myopic choroidal neovascularization, a vision-threatening complication of myopia (nearsightedness).

Byooviz is an anti-vascular endothelial growth factor (VEGF) therapy that prevents vision loss in patients with retinal vascular disorders, which can cause irreversible blindness or visual impairments in adults in the United States.

The FDA approval of Byooviz was based on a totality of evidence including analytical, nonclinical data, and clinical data. In a phase 3 study, the efficacy, safety, pharmacokinetics, and immunogenicity of Byooviz was compared with Lucentis in patients with wet AMD.

FDA approves Jakafi for GVHD.

The FDA has approved Incyte’s Jakafi (ruxolitinib) for treatment of chronic graft-versus-host disease (GVHD) after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.

Graft-versus-host disease is a systemic disorder that occurs when the graft's immune cells recognize the host as foreign and attack the recipient’s body cells. It can occur in up to half of patients receiving hematopoietic stem cell transplantation.

This approval is the fourth FDA-approved indication for Jakafi, which received FDA-approval in 2019 for steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

FDA approves topical JAK inhibitor for atopic dermatitis.

The FDA has approved Incyte’s Opzelura (ruxolitinib) cream for the short-term and non-continuous chronic treatment of mild-to-moderate atopic dermatitis in patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies. Opzelura is the first and only topical formulation of a JAK inhibitor approved in the United States. Research shows dysregulation of the JAK-STAT pathway contributes to key features of atopic dermatitis such as itch, inflammation, and skin barrier dysfunction.

Opzelura is a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib.

FDA approves Tivdak for advanced cervical cancer.

The FDA has granted accelerated approval to Genmab/Seagen’s Tivdak (tisotumab vedotin-tftv), the first approved antibody-drug conjugate (ADC) for the treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. Tivdak is approved under the FDA’s accelerated approval program based on tumor response and the durability of the response.

The approval is based on the phase 2 innovaTV 204 trial, which evaluated 101 patients.

Tivdak is an ADC composed of Genmab’s human monoclonal antibody directed to tissue factor and Seagen’s ADC technology that uses a protease-cleavable linker that covalently attaches the microtubule-disrupting agent monomethyl auristatin E to the antibody.

FDA approves Cabometryx for thyroid cancer.

The FDA has approved Exelixis’ Cabometyx (cabozantinib) for the treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer that has progressed following treatment with a vascular endothelial growth factor receptor (VEGFR)-targeted therapy and who are resistant to radioactive iodine treatment.

This is more than two months ahead of the Prescription Drug User Fee Act (PDUFA) target action date of December 4, 2021. DTC is the most common type of thyroid cancer in the United States, and patients who are resistant to radioactive iodine treatment face a poor prognosis.

FDA accepts BMS application for Opdivo/Relatimab combination for advanced melanoma.

The FDA has granted priority review of the Bristol Myers Squibb’s biologics license application (BLA) for the combination of relatlimab and Opdivo (nivolumab), administered as a single infusion, for the treatment of adult and adolescents with unresectable or metastatic melanoma. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 19, 2022.

The BLA submission was based on the efficacy and safety results of the phase 2/3 RELATIVITY-047 trial, which demonstrated that the combination therapy achieved progression-free survival benefit of 10.1 months compared with 4.6 months for Opdivo alone.

FDA has accepted application for rare epilepsy.

The FDA accepted for filing Marinus Pharmaceuticals’ new drug application (NDA) for the use of ganaxolone in the treatment of seizures associated with CDKL5 deficiency disorder, a rare, genetic epilepsy. The NDA was granted Priority Review designation and the FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of March 20, 2022.

The application is supported by data from the Marigold study, a phase 3 trial in 101 patients. Patients treated with ganaxolone showed a 30.7% median reduction in 28-day major motor seizure frequency, compared with a 6.9% reduction for those receiving placebo.

Patients in the open-label extension study treated with ganaxolone for at least 12 months experienced a median 49.6% reduction in major motor seizure frequency.

Ganaxolone received orphan drug designation in June 2017 and a Rare Pediatric Disease designation for CDKL5 deficiency disorder in July 2020.

AbbVie submits Skyrisi NDA for Crohn’s disease.

AbbVie has submitted an application to the FDA seeking approval for Skyrizi (risankizumab-rzaa), an interleukin-23 (IL-23) inhibitor, for the treatment of patients 16 years and older with moderate-to-severe Crohn’s disease. Approval is being sought for both the 600 mg intravenous induction and 360 mg subcutaneous maintenance therapy.

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