FDA updates for the week of August 8, 2022

In a busy week, the FDA 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 the monkeypox vaccine in children, expanded the review of omaveloxolone, established an advisory committee meeting for microbiota-based C. diff therapy and priority review of eleastrant in breast cancer. Additionally, Novaliq has submitted an NDA for the treatment of dry eye disease.

The FDA approves Enhertu for metastatic lung cancer.

The FDA has granted an expedited 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 antibody-drug conjugate (ADC) against HER2 jointly developed and marketed by Daiichi Sankyo and AstraZeneca.

The accelerated approval was based on 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 unresectable or metastatic non-squamous cell NSCLC with HER2 mutation that had received prior systemic therapy.

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

The FDA approves Myfembree to treat endometriosis.

The FDA has approved Myfembree (relugolix, estradiol and norethindrone acetate) as a single tablet, once-daily treatment 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 review of the additional new drug application for the endometriosis indication. Regulators want more time to review information they had requested from 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-responsive prostate cancer (mHSPC). Nubequa is an oral androgen receptor blocker that is also approved to treat non-metastatic castration-resistant prostate cancer.

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

The FDA approves the tablet form of Calquence.

The FDA has approved a Calquence tablet formulation of AstraZeneca (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 expedited approval based on overall response rate.

The approval was based on the results of a trial which showed that the capsule and tablet formulations of Calquence are bioequivalent, indicating that the same efficacy and safety profile can be expected with the same dosage and the same calendar. The tablet can be taken with stomach acid reducing agents including proton pump inhibitors, antacids and H2 receptor antagonists.

The FDA approves Xofluza to treat and prevent influenza 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 ages five to less than 12 years of age. Additionally, the FDA has approved Xofluza for the prevention of influenza in children ages five through 12 after contact with someone with influenza.

Xofluza is available as a single dose tablet. It is already FDA-approved to treat influenza in people 12 years of age and older who have had flu symptoms for less than 48 hours and who are otherwise healthy or at high risk of developing flu-related complications. flu. It is also approved to prevent influenza in people 12 years of age and older after contact with someone who has influenza.

Additionally, Xofluza is being studied in a Phase 3 development program, including children under one year of age, and to assess the potential to reduce direct transmission of influenza from otherwise healthy patients. health to family contacts.

The FDA issues an EUA for the monkeypox vaccine for those under 18.

The FDA has issued an Emergency Use Authorization (EUA) for the intradermal injection of Bavarian Nordic’s Jynneos monkeypox vaccine for people, including those under the age of 18, who are at high risk of monkeypox infection . This will increase the total number of doses available for use up to five times, the FDA said in a Press release. Monkeypox cases in the United States have climbed to 10,392 as of August 10, 2022, according to the CDCand HHS Secretary Xavier Becerra said the continued spread of the monkeypox virus in the United States was a Public Health Emergency (PHE).

Jynneos, the modified vaccinia ankara (MVA) vaccine, has been approved as a subcutaneous injection in 2019 for the prevention of smallpox and monkeypox in adults 18 years and older deemed to be at high risk of smallpox or monkeypox infection.

FDA extends review of omaveloxolone in Friedreich’s ataxia.

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

The company submitted updated data from the MOXIe Extension study, including a delayed-start analysis using a data cutoff of March 2022 and a new matched propensity analysis using patient data from clinical outcome measures in Friedreich’s ataxia study as controls. Additionally, Reata submitted an analysis of the relevance of Nrf2, the target of omaveloxolone, for the pathophysiology of Friedreich’s ataxia.

The PDUFA update date is February 28, 2023 and the scheduled advisory committee meeting is suspended pending review of the NDA changes.

FDA Hosts Microbiota Consultative Meeting C difference. therapy.

The FDA’s Vaccines and Related Biologics Advisory Committee (VRBPAC) will meet on September 22, 2022 to review data supporting Ferring Pharmaceutical’s Biologics License Application (BLA) to reduce It’s hard infection (CDI) after antibiotic treatment. RBX2660 is a microbiota-based live biotherapeutic that contains live microorganisms (bacteria or yeast) 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, it can lead to C difference.irritable bowel syndrome or diabetes. C difference. is a serious illness 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 that people who have had a recurrence are at significantly higher risk of subsequent infection.

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

FDA assigns priority review to elacestrant in metastatic breast cancer.

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

The NDA submission was supported by results 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 the ESR1 mutation. The data also showed a manageable safety profile.

Elacestrant is a Selective Estrogen Receptor Degrader (SERD), a 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 become resistant to tamoxifen or aromatase inhibitors.

Novaliq submits an NDA for the treatment of dry eye disease.

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

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

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