FDA Updates for Week of June 13, 2022

FDA expands EUA for Moderna, Pfizer-BioNTech COVID-19 vaccines to children 6 months and up.

After an FDA committee voted unanimously in favor of doing so, the FDA authorized emergency use of the Moderna COVID-19 vaccine and the Pfizer-BioNTech COVID-19 Vaccine for children down to 6 months of age.

The FDA amended the Moderna EUA for individuals 6 months through 17 years of age; it had been authorized for adults 18 years of age and older. It amended the Pfizer-BioNTech COVID-19 vaccine for people 6 months through 4 years of age. The vaccine had been authorized for use in people 5 years of age and older.

On June 15, 2022, the FDA’s Vaccines and Related Biological Products Advisory Committee unanimously voted to expand FDA's emergency use authorization (EUA) for Moderna’s COVID-19 vaccine for children ages 6 months to 5 years, and Pfizer’s vaccine for children ages 6 months to four years old.

In addition, the VRBPAC unanimously voted to approve Moderna’s COVID-19 vaccine for children ages 7 to 16 on June 14.

FDA approves Skyrizi for patients with Crohn’s disease.

The FDA has approved AbbVie’s Skyrizi (risankizumab-rzaa) as the first specific interleukin-23 (IL-23) inhibitor for the treatment of adults with moderate-to-severe Crohn’s disease.

The approval was based on two induction and one maintenance clinical trials, in which Skyrizi demonstrated significant improvements in endoscopic response and clinical remission compared with placebo, as both an induction and maintenance therapy.

The dosing regimen is 600 mg administered by intravenous infusion over at least one hour at week 0, week 4, and week 8, followed by 360 mg self-administered by subcutaneous injection with an on-body injector at week 12, and every 8 weeks thereafter. A 180 mg self-administered subcutaneous maintenance dose option remains under review by the FDA.

FDA approves Olumiant to treat patients with alopecia.

The FDA has approved Eli Lilly’s Olumiant (baricitinib) oral tablets to treat adult patients with severe alopecia, a disorder that often appears as patchy baldness. This is the first FDA approval of a systemic treatment for alopecia, which affects more than 300,000 people in the United States each year.

Alopecia is an autoimmune disorder in which the body attacks its own hair follicles, causing hair to fall out, often in clumps. Olumiant is a Janus kinase (JAK) inhibitor, which blocks the activity of one or more of a specific family of enzymes, interfering with the pathway that leads to inflammation.

Olumiant is a once-daily, oral JAK inhibitor that was discovered by Incyte and licensed to Lilly. It is approved in the United States and more than 75 countries as a treatment for adults with moderate to severe rheumatoid arthritis.

The labeling for Olumiant includes a boxed warning for risk of serious infections, mortality, malignancy, major adverse cardiovascular events (MACE) and thrombosis. In December 2021, the FDA required an updated boxed warning on the three top-selling JAK inhibitors — Pfizer’s Xeljanz Olumiant, and AbbVie’s Rinvoq (upadacitinib) — used to treat rheumatoid arthritis and other inflammatory conditions.

FDA approves Imcivree for obesity associated with the rare disease BBS.

The FDA has approved Rhythm Pharmaceuticals’ supplemental new drug application (sNDA) for Imcivree (setmelanotide) for patients with Bardet-Biedl syndrome (BBS). It is indicated for chronic weight management in adults and children 6 years old and older with obesity in patients with BBS.

Bardet Biedl Syndrome is a rare genetic disorder that impacts about 1,500 to 2,500 people in the United States. It can have variable symptoms that include retinal degeneration, obesity, reduced kidney function, or extra fingers or toes. Weight is generally normal at birth but infants with BBS quickly gain weight, with fat distributed in the abdomen and chest. About 90% of patients develop obesity. This can lead to diabetes and complications related to the heart and blood vessels.

The approval was based on data from a phase 3 trial of 31 patients where Imcivree met primary and secondary endpoints. In patients six years and older, the mean change in BMI was -7.9% without diet or exercise, and there was a statistically significant change in hunger score.

At the same time, the FDA issued a complete response letter for Imcivree for use in patients with Alstrom syndrome, another genetic rare disease that can cause obesity from a mutation in the ALMS gene.

FDA approves Amvuttra for neuropathy associated with rare disease.

The FDA has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease caused by mutations of the transthyretin (TTR) gene. In hATTR, amyloid proteins build up the heart, kidneys, liver and other organs. Generally, patients first present with neuropathy, damage to nerves that results in numbness and muscle weakness. In addition to polyneuropathy, hATTR amyloidosis can lead the loss of the ability to walk unaided, a reduced quality of life, and a decline in cardiac functioning.

FDA extends PDUFA date for sNDA of Brukinsa.

The FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date by three months to Jan. 20, 2023 for the supplementary new drug application (sNDA) for BeiGene’s Brukinsa (zanubrutinib). The FDA is reviewing the therapy as a treatment for adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma.

The FDA extended the PDUFA date to allow time to review additional clinical data submitted by BeiGene. This includes the final response analysis from the global ALPINE clinical trial. The trial enrolled a total of 652 patients who received either Brukinsa or Imbruvica (ibrutinib) until disease progression or unacceptable toxicity. Imbruvica is jointly developed by Janssen and Pharmacyclics, an AbbVie company.

Brukinsa is approved for three indications in the United States: for the treatment of mantle cell lymphoma in adult patients who have received at least one prior therapy (November 2019); for the treatment of adult patients with Waldenström’s macroglobulinemia (August 2021); and for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (September 2021).

FDA accepts Keytruda application for earlier NSCLC after surgery.

The FDA has accepted for review Merck’s supplemental biologics license application (sBLA) seeking approval for Keytruda (pembrolizumab) for the adjuvant treatment of patients with stage IB, II or IIIA non-small cell lung cancer (NSCLC) following complete surgical resection.

The FDA has set a Prescription Drug User Fee Act (PDUFA) date of Jan. 29, 2023. But Merck indicated in a press release that additional data may be provided during the review process that may delay this date.

The sBLA is based on data from the pivotal phase 3 KEYNOTE-091 trial, which was conducted in collaboration with the European Organisation for Research and Treatment of Cancer (EORTC) and the European Thoracic Oncology Platform (ETOP). The study has dual primary endpoints of disease-free survival in both patients with and without PD-L1 tumor expression.