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FDA Updates for the Week of August 8, 2022


In a busy week, the FDA has approved additional indications for Enhertu, Myfembree and Nubeqa, as well as a tablet form of Calquence. The agency also approved Xofluza for children, issued an EUA for monkeypox vaccine in children, extended the review of omaveloxolone, set up advisory committee meeting for microbiota-based C. diff therapy, and priority review for elacestrant in breast cancer. Additionally, Novaliq submited NDA for dry eye treatment.

FDA approves Enhertu for metastatic lung cancer.

The FDA has granted accelerated approval for Enhertu (fam-trastuzumab deruxtecan-nxki) to treat adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have HER2 mutations. Enhertu is an HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.

The accelerated approval was based on the results from the DESTINY-Lung02 phase 2 trial. An interim efficacy analysis in a pre-specified patient cohort showed that Enhertu demonstrated a confirmed objective response rate of 57.7% in patients with HER2 mutant unresectable or metastatic non-squamous NSCLC who had received one prior systemic therapy.

The FDA also approved Life Technologies’s Oncomine Dx Target Test (tissue) and the Guardant Health’s Guardant360 CDx (plasma) as companion diagnostics for Enhertu.

FDA approves Myfembree to treat endometriosis.

The FDA has approved Myfembree (relugolix, estradiol, and norethindrone acetate) as a one-pill, once-a-day therapy for the management of moderate-to-severe pain associated with endometriosis. Developed by Myovant Sciences and Pfizer, Myfembree is also approved for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women.

In May 2022, the FDA had extended the review of the supplemental new drug application for the endometriosis indication. Regulators want additional time to review information they had requested from the companies regarding bone mineral density.

FDA approves additional indication for Nubeqa.

The FDA has approved a supplemental new drug application (sNDA) for Bayer’s Nubeqa (darolutamide) with docetaxel for the treatment of adult patients with metastatic hormone-sensitive prostate cancer (mHSPC). Nubequa is oral an androgen receptor inhibitor that is also approved to treat non-metastatic castration-resistant prostate cancer.

The approval was made under the FDA’s Real-Time Oncology Review (RTOR) pilot program based on results of the phase 3 ARASENS trial. Results demonstrated a statistically significant increase in overall survival (OS), the trial’s primary endpoint, with a reduction in the risk of death by 32%.

FDA approves tablet form of Calquence.

The FDA has approved a tablet formulation of AstraZeneca’s Calquence (acalabrutinib) for all current indications, including adult patients with chronic lymphocytic leukemia, small lymphocytic lymphoma and for patients with relapsed or refractory mantle cell lymphoma, which is approved under accelerated approval based on overall response rate.

The approval was based on results from a trial that showed the Calquence capsule and tablet formulations are bioequivalent, indicating the same efficacy and safety profile can be expected with the same dosing strength and schedule. The tablet can be taken with gastric acid-reducing agents, including proton pump inhibitors, antacids and H2-receptor antagonists.

FDA approves Xofluza to treat and prevent flu in children.

The FDA has approved a supplemental new drug application (sNDA) for Genentech’s Xofluza (baloxavir marboxil) for the treatment of acute uncomplicated influenza in children aged five to less than 12 years of age. Additionally, the FDA approved Xofluza for the prevention of influenza in children aged five to less than 12 years of age following contact with someone with influenza.

Xofluza is available as a one-dose, single tablet. It is already FDA-approved to treat influenza in people 12 years of age and older who have had influenza symptoms for no more than 48 hours and who are otherwise healthy or at high risk of developing influenza-related complications. It is also approved to prevent influenza in people 12 years of age and older following contact with someone with influenza.

Additionally, Xofluza is being studied in a phase 3 development program, including children under the age of one as well as to assess the potential to reduce direct transmission of influenza from otherwise healthy patients to household contacts.

FDA issues EUA for monkeypox vaccine for those under 18.

The FDA issued an emergency use authorization (EUA) for the Bavarian Nordic’s Jynneos monkeypox vaccine intradermal injection for people, including those under the age of 18, who are at high risk for monkeypox infection. This will increase the total number of doses available for use by up to five-fold, FDA said in a news release. U.S. monkeypox cases have soared to 10,392 as of Aug. 10, 2022, according to the CDC, and HHS Secretary Xavier Becerra declared the ongoing spread of monkeypox virus in the United States a Public Health Emergency (PHE).

Jynneos, the modified vaccinia ankara (MVA) vaccine, was approved as a subcutanous injection in 2019 for prevention of smallpox and monkeypox disease in adults 18 years of age and older determined to be at high risk for smallpox or monkeypox infection.

FDA extends review of omaveloxolone in Friedreich’s ataxia.

The FDA has extended by three months the review of Reata Pharmaceuticals’ omaveloxolone for the treatment of patients with Friedreich’s ataxia, a rare, genetic, degenerative neuromuscular disorder.

The company has submitted updated data from the MOXIe Extension study, including delayed-start analysis using a March 2022 data cut-off and a new propensity-matched analysis using patient data from the Clinical Outcome Measures in Friedreich’s Ataxia Study as controls. Additionally, Reata submitted an analysis of the relevance of Nrf2, the target of omaveloxolone, to the pathophysiology of Friedreich’s ataxia.

The updated PDUFA date is Feb. 28, 2023, and the planned advisory committee meeting is on hold pending review of NDA amendments.

FDA schedules advisory meeting for microbiota-based C. diff therapy.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) will hold a meeting on Sept. 22, 2022, to review data supporting Ferring Pharmaceutical’s biologics license application (BLA) to reduce recurrent C. difficile infection (CDI) after antibiotic treatment. RBX2660 is microbiota-based live biotherapeutic that contains live microorganisms (bacteria or yeast) that are used as active substances.

The human microbiome is a complex community of microorganisms in and on the body. In the gut, if a microbial imbalance occurs, this can lead to C. diff, irritable bowel syndrome or even diabetes. C. diff is a serious disease that causes severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea, and colitis. It has been estimated that up to 35% of cases recur after initial diagnosis and people who have had a recurrence are at significantly higher risk of further infection.

The FDA has granted RBX2660 fast track, orphan, and breakthrough therapy designations. RBX2660 was developed by Rebiotix, a Ferring company.

FDA assigns priority review for elacestrant in metastatic breast cancer.

The FDA has accepted the Menarini’s new drug application (NDA) for elacestrant to treat patients with ER+/HER2 advanced or metastatic breast cancer. The FDA has granted the application priority review and assigned a PDUFA date of Feb. 17, 2023. The NDA was submitted in June 2022.

The NDA submission was supported by results of the phase 3 data from the EMERALD study, which showed that elacestrant reduced the risk of disease progression or death by 30% in all patients and by 45% in patients with ESR1 mutation. The data also showed a manageable safety profile.

Elacestrant is a selective estrogen receptor degrader (SERD), type of drug that down regulates the estrogen receptor. SERD and full receptor antagonists are essential treatment options for HR-positive breast cancer, especially in patients who have been become resistant to tamoxifen or aromatase inhibitors.

Novaliq submits NDA for dry eye treatment.

Novaliq has submitted a new drug application (NDA) to the FDA for CyclASol (cyclosporine ophthalmic solution) to treat patients dry eye disease (DED). CyclASol is a preservative-free, water-free, anti-inflammatory and immunomodulating ophthalmic solution, containing 0.1% cyclosporine. While it is not water-soluble, cyclosporine can be soluble in the excipient perfluorobutylpentane using Novaliq’s EyeSol technology. The technology allows for improved bioavailability and better efficacy.

The NDA is supported by safety and efficacy results in more than 1,000 patients with dry eye disease from a phase 2 dose finding study, the phase 2b/3 ESSENCE-1 study, the phase 3 ESSENCE-2 study and its open label extension.

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