FDA Updates for the Week of Aug. 15, 2022


In COVID-19 news, Novaxax seeks EUA for vaccine booster. The FDA has approved Bluebird’s $2.8 million gene therapy for blood disease, as well a high-concentration of Hadlima, a Humira biosimilar. The agency has also PDUFA for several products, including Lynparza/abiraterone prostate cancer, Polivy for a blood cancer, and fezolinetant for menopause.

FDA approves gene therapy for blood disease.

The FDA has approved Bluebird bio’s Zynteglo (betibeglogene autotemcel), also known as beti-cel, the first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Bluebird bio has set wholesale acquisition cost of $2.8 million for Zynteglo and also has set up an outcomes-based contract offering that includes a single upfront payment and up to 80% risk-sharing. Zynteglo will be available through a network of qualified treatment centers with experience delivering novel cell and gene therapies

Beta-thalassemia is a severe genetic blood disease caused by mutations in the beta-globin gene and is characterized by significantly reduced or absent adult hemoglobin production. This can result in severe anemia and lifelong dependence on red blood cell transfusions.

Bluebird bio’s outcomes program includes a guarantee to reimburse contracted commercial and government payers up to 80% of the cost of the therapy if a patient fails to achieve and maintain transfusion independence up to two years following infusion. In phase 3 trials, all patients who achieved transfusion independence have remained transfusion free, the company said in a press release.

FDA approves high concentration Hadlima.

The FDA has approved a high-concentration (100 mg/mL) formulation of Hadlima (adalimumab-bwwd), a biosimilar referencing AbbVie’s Humira (adalimumab). The FDA had approved Hadlima in a low-concentration (50 mg/mL) formulation in July 2019. The biosimilar will be available in pre-filled syringe and autoinjector options when it launches July 1, 2023.

Hadlima was developed by South Korea-based Samsung Bioepis and will be commercialized by Organon.

Outside the United States, the low concentration formulation of the biosimilar has been available since 2018. Hadlima is a tumor necrosis factor (TNF) blocker used to treat rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, Crohn’s disease and ankylosing spondylitis.

The FDA approval of high-concentration Hadlima was based on clinical data from a randomized, single-dose study that compared the pharmacokinetics, safety, tolerability, and immunogenicity of the two formulations in healthy volunteers.

FDA grants priority review for Lynparza/abiraterone combo for metastatic prostate cancer.

The FDA has accepted and granted priority review of a supplemental new drug application (sNDA) for Lynparza (olaparib) in combination with abiraterone and prednisone or prednisolone to treat adult patients with metastatic castration-resistant prostate cancer (mCRPC). The FDA has set a Prescription Drug User Fee Act (PDUFA) date in the fourth quarter of 2022.

Lynparza is a first-in-class PARP inhibitor developed by Merck and AstraZeneca. It is the first targeted treatment to potentially exploit DNA damage response pathway deficiencies, such as BRCA mutations.

The application is based on the phase 3 PROpel trial, in which the Lynparza combination reduced the risk of disease progression or death by 34% compared with placebo plus abiraterone and prednisone or prednisolone. Median radiographic progression-free survival (rPFS) was 24.8 months for the Lynparza arm versus 16.6 months for the placebo arm. About 14% of patients who received the combination treatment discontinued treatment due to an adverse event.

FDA accepts Polivy sBLA for aggressive blood cancer.

The FDA has accepted Genentech’s supplemental biologics license application (sBLA) for Polivy (polatuzumab vedotin-piiq) in combination with monoclonal antibody Rituxan (rituximab) plus cyclophosphamide, doxorubicin and prednisone (R-CHP) for the treatment of people with previously untreated diffuse large B-cell lymphoma (DLBCL). The FDA is expected to make a decision on approval by April 2, 2023.

DLBCL is a cancer that starts in white blood cells, specifically those that make antibodies to fight infections. Polivy is a first-in-class antibody-drug that targets CD79b, which is expressed on the surface of malignant B cells. The FDA approved Polivy in June 2019 to be used in combination with the chemotherapy drug bendamustine and rituximab for adult patients with relapsed or refractory diffuse large B-cell lymphoma. Currently, Polivy is used off label with R-CHP combination.

This application is based on the results from the pivotal phase 3 POLARIX trial, which is the first in two decades to show a clinically meaningful improvement in progression-free survival (PFS) compared with the current standard of care Rituxan plus cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP).

The risk of disease progression, relapse or death was reduced by 27% with Polivy plus R-CHP compared with R-CHOP after a median follow-up of 28.2 months.

FDA to review NDA for nonhormonal therapy for menopause.

The FDA has accepted Astellas Pharma’s new drug application (NDA) for fezolinetant. Astellas seeking approval to treat moderate-to-severe vasomotor symptoms (VMS) associated with menopause, which is characterized by hot flashes and/or night sweats. The PDUFA target action date is Feb. 22, 2023, following use of a priority review voucher. Astellas proposes a 45 mg daily dose.

Fezolinetant is an oral, nonhormonal therapy that works by blocking neurokinin B (NKB) binding on the KNDy neuron to moderate neuronal activity in the hypothalamus to reduce the frequency and severity of symptoms associated with menopause.

The NDA was submitted in June 2022. The application is supported by results from the BRIGHT SKY program, which included three phase 3 clinical trials that collectively enrolled more than 2,800 women with VMS across the United States, Canada and Europe.

Novaxax seeks EUA for COVID-19 booster.

Novavax has submitted an application to the FDA for emergency use authorization (EUA) for its protein-based COVID-19 vaccine booster in adults 18 years or older.

This application is supported by data from Novavax’s phase 3 PREVENT-19 trial conducted in the United States and Mexico, and from the UK-sponsored COV-BOOST phase 2 trial. As part of an open-label booster phase of the PREVENT-19 trial, a single booster dose of the Novavax COVID-19 Vaccine, Adjuvanted, was administered to healthy adult participants at least six months after their primary two-dose vaccination series.

The third dose produced robust antibody responses comparable to or exceeding levels associated with the efficacy data in the primary series phase 3 clinical trials. In the COV-BOOST trial, the vaccine induced a significant antibody response when used as a third booster dose. In the PREVENT-19 trial, following the booster, local and systemic reactions had a median duration of approximately two days

Last month, the FDA granted an EUA for the primary series of the vaccine. This followed the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) positive vote to recommend the FDA grant EUA for the vaccine.

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