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FDA News for the Week of Sept. 27, 2021


Approvals include a therapy for a rare disease for children and a novel migraine prevention. New regulatory applications include Biogen’s second Alzheimer’s therapy and other indications for Opdivo and Erbitux, and Pfizer submits data for COVID-19 vaccine for children.

The FDA approves Repatha for children with rare genetic disease.

The FDA has approved Amgen’s Repatha (evolocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH).

HeFH is an inherited, genetic condition with a prevalence of one in 250 people worldwide. High levels of LDL-C starting at birth accelerate the development of atherosclerotic cardiovascular disease, leading to an overall increased risk of cardiovascular events, including heart attack and other vascular conditions, at an earlier age.

FDA approves Erbitux/Braftovi combination for advanced colorectal cancer.

The FDA has granted approval of a new indication for Lilly’s Erbitux (cetuximab) in combination with Pfizer’s Braftovi (encorafenib) for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation.

Erbitux is the first anti-EGFR antibody approved for this indication and is based on results from Pfizer’s BEACON CRC trial, a phase 3 trial to specifically study patients with previously treated metastatic CRC with a BRAF V600E mutation. With this approval, Erbitux has now received seven FDA approvals to treat certain types of CRC and squamous cell carcinoma of the head and neck.

Colorectal cancer is the fourth most common type of cancer, according to the National Cancer Institute. The NCI estimates that in 2021, there will be almost 150,000 new cases of colorectal cancer and more than 50,000 deaths.

FDA approves novel therapy to prevent migraine.

The FDA has approved AbbVie’s Qulipta (atogepant) for the prevention of migraine in adults. The therapy is the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist specifically developed for the prevention of migraine.

The approval is based on data from a clinical trial program that evaluated Qulipta in almost 2,000 patients who experienced four to 14 migraine days per month, including the pivotal phase 3 ADVANCE study — which was published in The New England Journal of Medicine in August 2021— the pivotal phase 2b/3 study, and the phase 3 long-term safety study.

Biogen/Eisai submits rolling application for second Alzheimer’s therapy.

Biogen and Eisai have initiated a rolling submission to the FDA of a biologics license application (BLA) for lecanemab, an anti-amyloid beta protofibril antibody, for the treatment of early Alzheimer’s disease. The application is being submitted under the accelerated approval pathway based on a trial that uses beta amyloid reduction as a surrogate end point.

A phase 2b clinical trial in people with early Alzheimer’s disease and confirmed amyloid pathology showed lecanemab lowered beta amyloid plaque and reduced clinical decline across several clinical endpoints.

The results of this study were published in April 2021. This study was designed as an 18-month, proof-of-concept study. The study explored the dose response of lecanemab over three dose levels and two dosing regimens with the objective to establish the most effective (ED90) dose of lecanemab.

Investigators found, however, that lecanemab did not meet the primary end point, which was a 12-month clinical change on the Alzheimer’s Disease Composite Score. Investigators did find that on the 18-month Bayesian and other analyses, lecanemab demonstrated reduction in brain amyloid accompanied by a consistent reduction of clinical decline across several clinical and biomarker end points based on the Alzheimer’s Disease Composite Score.

A phase 3 confirmatory study continues. This study completed enrollment in March 2021.

FDA accepts applications for Opdivo combinations for advanced esophageal squamous cell carcinoma.

The FDA has accepted the Bristol Myers Squibb’s supplemental biologics license applications for both Opdivo (nivolumab) in combination with Yervoy (ipilimumab) and Opdivo in combination with fluoropyrimidine- and platinum-containing chemotherapy as first-line treatments for adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC).

The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 28, 2022.

Pfizer and BioNTech submit initial data of COVID-19 vaccine in children.

Pfizer and BioNTech have submitted data to the FDA from the phase 2/3 trial of their COVID-19 vaccine in children 5 to <12 years of age.

The companies announced positive topline results from the pivotal trial on Sept. 20, 2021. In the trial, which included 2,268 participants 5 to less than 12 years of age, the vaccine demonstrated a favorable safety profile and elicited antibody responses using a two-dose regimen of 10 μg doses. These results were comparable to those recorded in a previous Pfizer-BioNTech study in people 16 to 25 years of age, who were immunized with 30 μg doses. The 10 μg dose was selected as the preferred dose for safety, tolerability and immunogenicity in children 5 to less than 12 years of age.

FDA accepts application for Libtayo for advanced cervical cancer.

The FDA has accepted for priority review Regeneron Pharmaceuticals’ supplemental biologics license application (sBLA) for PD-1 inhibitor Libtayo (cemiplimab-rwlc) to treat patients with recurrent or metastatic cervical cancer whose disease progressed on or after chemotherapy. The target action date for the FDA decision is Jan. 30, 2022.

The sBLA is also being reviewed under the FDA’s Project Orbis initiative, which will allow for concurrent review by participating health authorities in Australia, Brazil, Canada, and Switzerland. Additional global regulatory submissions are planned, including in the European Union by the end of 2021.

The sBLA is supported by results from a phase 3 trial that enrolled patients irrespective of

PD-L1 expression status and is being conducted with The GOG Foundation, the European Network for Gynaecological Oncological Trial groups, and NRG Oncology-Japan. Results were first presented as part of a European Society for Medical Oncology (ESMO) Virtual Plenary in May 2021.

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