Best news from the FDA for the week of September 20, 2021
The FDA authorized the booster to be administered at least six months after the end of the primary vaccination course.
FDA approves first biosimilar to treat macular degeneration.
The FDA has approved Biogen and Samsung’s Byooviz Bioepis (ranibizumab-nuna) as the first biosimilar to Lucentis from Genentech (ranibizumab) for the treatment of several eye diseases and conditions, including age-related neovascular (wet) macular degeneration , a major cause of vision loss and blindness for Americans over 65. Byooviz is also approved to treat macular edema after retinal venous 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) treatment that prevents vision loss in patients with retinal vascular disorders, which can cause irreversible blindness or visual impairment in adults in the United States.
The FDA’s approval of Byooviz was based on a body of evidence, including analytical, non-clinical and clinical data. In a phase 3 study, the efficacy, safety, pharmacokinetics and immunogenicity of Byooviz were compared with those of Lucentis in patients with wet AMD.
FDA approves Jakafi for GVHD.
The FDA has approved Jakafi (ruxolitinib) of Incyte for the 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 of age and more.
Graft-versus-host disease is a systemic disorder that occurs when the transplant’s immune cells recognize the host as foreign and attack cells in the recipient’s body. It can occur in up to half of patients receiving a hematopoietic stem cell transplant.
This approval is the fourth FDA-approved indication for Jakafi, which received FDA approval in 2019 for acute steroid refractory GVHD in adult and pediatric patients 12 years of age and older.
The FDA approves topical JAK inhibitor for atopic dermatitis.
The FDA has approved Opzelura (ruxolitinib) cream from Incyte for the short-term, non-continuous chronic treatment of mild to moderate atopic dermatitis in patients 12 years of age and older whose disease is not adequately controlled by topical prescription treatments. Opzelura is the first and only topical formulation of a JAK inhibitor approved in the United States. Research shows that deregulation of the JAK-STAT pathway contributes to key features of atopic dermatitis such as itching, inflammation, and skin barrier dysfunction.
Opzelura is a new cream formulation of ruxolitinib, a selective JAK1 / JAK2 inhibitor of Incyte.
FDA approves Tivdak for advanced cervical cancer.
FDA Granted Fast-Track Approval for Genmab / Seagen’s Tivdak (tisotumab vedotin-tftv), the First Antibody-Drug Conjugate (ADC) Approved for the Treatment of Adult Patients with Recurrent or Metastatic Cervical Cancer with disease progression during or after chemotherapy. Tivdak is approved under the FDA’s Fast Track Approval Program based on tumor response and durability of response.
The approval is based on the innovaTV 204 phase 2 trial, which evaluated 101 patients.
Tivdak is an ADC composed of Genmab’s human monoclonal antibody to tissue factor and Seagen’s ADC technology that utilizes a protease-cleavable linker that covalently attaches the microtubule disruptor monomethyl auristatin E to antibody.
The FDA approves Cabometryx for thyroid cancer.
The FDA has approved Cabometyx (cabozantinib) from Exelixis 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 after treatment with drug-targeted therapy. vascular endothelial growth factor receptor (VEGFR). and which are resistant to radioactive iodine treatment.
This is more than two months before the Prescription Drug User Fee Act (PDUFA) target action date of December 4, 2021. DTP is the most common type of thyroid cancer in the United States, and patients resistant to radioactive iodine treatment face a poor prognosis.
The FDA accepts BMS’s request for the combination Opdivo / Relatimab for advanced melanoma.
The FDA has granted priority review of 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 adults and adolescents with unresectable or metastatic melanoma. The FDA has set the Prescription Drug User Fee Act (PDUFA) deadline for 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 provided a progression-free survival benefit of 10.1 months against 4.6 months for Opdivo alone.
The FDA accepted the request for rare epilepsy.
The FDA has agreed to file 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 has been granted the priority review designation and the FDA has assigned an action date under the Prescription Drug User Fee Act (PDUFA) of March 20, 2022.
The claim is supported by data from the Marigold study, a Phase 3 trial in 101 patients. Patients treated with ganaxolone had a median reduction of 30.7% in the frequency of major motor seizures over 28 days, compared with a reduction of 6.9% for those receiving placebo.
Patients in the open-label extension study treated with ganaxolone for at least 12 months experienced a median reduction of 49.6% in the frequency of major motor seizures.
Ganaxolone received orphan drug designation in June 2017 and 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 for approval of Skyrizi (risankizumab-rzaa), an interleukin-23 (IL-23) inhibitor, for the treatment of patients 16 years of age and older with Moderate to severe Crohn’s. Approval is sought for 600 mg intravenous induction therapy and 360 mg subcutaneous maintenance therapy.