FDA Updates for the Week of June 5, 2023


The FDA has made three approvals this week: a novel drug for dry eye disease, a new indication for Prevymis to prevent CMV disease in adult kidney transplant recipients, and another prostate cancer indication for Lynparza. The agency has also set target action dates for the first CRISPR gene edited therapy and a supplement indication for Jemperli for earlier treatment of endometrial cancer. Additionally, Janssen submitted sBLA for Carvykti for early treatment of myeloma.

FDA Approves Novel Cyclosporine for Dye Eye Disease

The FDA has approved Novaliq’s Vevye (cyclosporine) 0.1% to treat patients with dry eye disease. Vevye (which had development name CyclASol) is a water-free, preservative-free solution based on EyeSol technology. Cyclosporine is not water-soluble, but the EyeSol excipient perfluorobutylpentane allows for improved bioavailability and better efficacy on the target tissue. The product contains no oils, no surfactants and is preservative-free due to the novel carrier.

Additionally, last month, Novaliq and development partner Bausch + Lomb announced the FDA approval for a different dry therapy: Miebo (perfluorohexyloctane; formerly known as NOV03). Miebo is the first treatment for dry eye that directly targets tear evaporation. A leading cause of DED is excessive tear evaporation, which due to an altered tear lipid layer, is often associated with the clinical signs of Meibomian gland dysfunction (MGD).

Company executives said Miebo will be available in the second half of 2023.

FDA Approves Prevymis for CMV Prevention in Kidney Transplant Patients

The FDA has approved a new indication for Merck’s Prevymis (letermovir) to prevent cytomegalovirus (CMV) disease in adult kidney transplant recipients at high risk. Prevymis is an antiviral agent that was initially approved by the FDA in 2017 to prevent CMV infection and disease in adult patients who have received allogeneic hematopoietic stem cell transplant. Prevymis is administered once-daily as an oral tablet or as an injection for intravenous infusion.

The retail price of Prevymis starts at $6,925.78 for 28 tablets of the 480 mg strength, according to GoodRx. Merck offers a coupon for privately insured patients to receive Prevymis at $15 per prescription for up to four prescriptions. Maximum savings is $2,500 per prescription.

FDA Clears Lynparza for Prostate Cancer with High Mortality Risk

The FDA granted another prostate cancer indication to AstraZeneca and Merck’s Lynparza (olaparib). It is now approved in combination with abiraterone and prednisone or prednisolone for patients with BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC).

Lynparza is already approved in the United States as monotherapy for patients with homologous recombination repair (HRR) gene-mutated mCRPC who have progressed following prior treatment with enzalutamide or abiraterone. The wholesale acquisition cost (WAC) of a 150 mg tablet, 30-day supply of Lynparza is $15,886.92.

FDA Sets PDUFA Dates for First CRISPR Gene Edited Therapy

The FDA has accepted Vertex Pharmaceuticals’ biologics license applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The FDA has granted priority review for the sickle cell indication and standard review for the beta thalassemia indication.

The agency has assigned Prescription Drug User Fee Act (PDUFA) target action dates of Dec. 8, 2023, for the sickle cell application and March 30, 2024, for the beta thalassemia application.

Exa-cel is a one-time therapy that uses a patient’s own hematopoietic stem cells that are edited to produce high levels of fetal hemoglobin in red blood cells. Increasing fetal hemoglobin has the potential to reduce or eliminate vaso-occlusive crisis — a condition tissues become deprived of oxygen, which causes an inflammatory response — for patients with sickle cell and transfusion requirements for patients with beta thalassemia.

The gene editing technique, CRISPR/Cas9, was developed by CRISPR Therapeutics. Vertex has collaborated with CRISPR Therapeutics for the development of exa-cel, formerly known as CTX001. Emmanuelle Charpentier, Ph.D., one of CRISPR Therapeutics’ scientific founders, co-invented CRISPR/Cas9 gene editing. Charpentier and Jennifer A. Doudna, Ph.D., won a Nobel Prize in Chemistry in 2020. CRISPR/Cas9 acts like a “genetic scissors” precisely cutting DNA and then allowing natural DNA repair processes to take over.

FDA to Review sBLA for Jemperli for Earlier Treatment of Endometrial Cancer

The FDA has accepted GSK’s supplemental biologics license application (sBLA) for Jemperli (dostarlimab). The application seeks approval of Jemperli in combination with chemotherapy for the treatment of adult patients with primary advanced or recurrent endometrial cancer. It would be indicated for patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H). If approved, this would be the first frontline treatment advancement in decades for patients with primary advanced or recurrent endometrial cancer.

The FDA granted priority review for this application and assigned a Prescription Drug User Fee Act action date of Sept. 23, 2023.

In February, Jemperli was granted full approved as monotherapy in dMMR recurrent or advanced endometrial cancer that has progressed on or following a prior platinum-containing regimen. The price of Jemperli is about $11,540 per 500 mg/10 mL vial, which is initially given once every three weeks for four doses, according to Drugs.com. GSK offers co-pay assistance of up of $26,000 for patients with commercial insurance.

Janssen Submits sBLA for Carvykti for Early Treatment of Myeloma

Janssen is seeking approval of a new indication for Carvytki (ciltacabtagene autoleucel; cilta-cel) to treat adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. The submission is based on data from the phase 3 CARTITUDE-4 study. Carvytki is a CAR-T cell therapy that Janssen licensed from Legend Biotech.

The FDA had approved Carvykti in February 2022 to treat adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy. Carvytki has a list price of $504,344 for a one-time infusion, according to Drugs.com.

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