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FDA Updates for the Week of Oct. 2, 2023


The FDA approved the first biosimilar of the arthritis medication Actemra, as well as plaque psoriasis drug Zoryve for children. The agency issued a complete response letter for lebrikizumab in atopic dermatitis and for liquid formulation of botulinum toxin for frown lines, and assigned a review date for a gene therapy for a rare immune disorder. Additionally, Takeda plans to withdraw the oncology drug Exkivity from the market while Coherus resubmits BLA for Udenyca OnBody.

Oncology Drug Exkivity to be Withdrawn from U.S. Market

Takeda is voluntarily withdrawing Exkivity (mobocertinib) from the U.S. market because a phase 3 confirmatory study did not meet its primary endpoint. Exkivity was granted accelerated approval in September 2021 to treat adult patients with advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

Exkivity remains available while Takeda works with the FDA on withdrawal timing, and the company said that patients currently taking the therapy will continue to have access after it is withdrawn. Takeda also plans to initiate a similar withdrawal globally where Exkivity approved and is working with regulators in other countries where it is currently available on next steps.

FDA Approves First Biosimilar of Arthritis Med Actemra

The FDA has approved Biogen’s Tofidence (tocilizumab-bavi) as first biosimilar to reference Genentech’s Actemra (tocilizumab). Tofidence is an interleukin-6 (IL-6) receptor antagonist that targets specific inflammatory proteins to suppress the immune system.

Tofidence is approved to treat rheumatoid arthritis in adults, polyarticular juvenile idiopathic arthritis in patients ages 2 and older, and systemic juvenile idiopathic arthritis in patients ages 2 and older. Tofidence, previously known as BIIB800, is administered via intravenous infusion.

Developed by Bio-Thera, Tofidence will be commercialized by Biogen in the United States. Biogen is still evaluating launch timeline, and information on pricing will be available closer to launch

Zoryve is Now Approved for Children with Plaque Psoriasis

FDA has approved the supplemental new drug application (sNDA) for Zoryve (roflumilast) cream 0.3% for the topical treatment of plaque psoriasis in children 6 to 11 years of age. Developed by Arcutis Biotherapeutics, Zoryve is a once-daily, steroid-free cream. This approval expands Zoryve indications; it was approved in July 2022 to treat those 12 years and older for the same indication.

The dosage for children 6 to 11 is the same as for those over the age of 12 and pricing is same for both pediatrics and adults. Zoryve’s wholesale acquisition cost is $825 per tube. Those with commercial drug insurance may be eligible for the Zoryve Direct Savings Card. Patients may pay as little as $25 if their commercial drug insurance covers. Individual patient savings are limited to $875 per fill and $5,000 in maximum total savings per calendar year.

FDA Issues CRL for Lebrikizumab in Atopic Dermatitis

The FDA has issued a complete response letter for Lilly’s biologic license application (BLA) for lebrikizumab to treat patients with moderate-to-severe atopic dermatitis. At issue are findings from the agency’s inspection of a third-party manufacturing company. The FDA indicated there were no concerns about the safety or labeling of lebrikizumab.

Lebrikizumab is monoclonal antibody that inhibits IL-13. The FDA submission was based on data from several trials, including ADvocate 1, ADvocate 2 and ADhere studies, which assess patients 12 years and older with moderate-to-severe eczema.

Manufacturing Issues Lead to CRL for Liquid Botulinum Toxin

The FDA has issued a complete response letter for Galderma’s biologics licensing application relabotulinumtoxinA (QM-1114) to treat patients with moderate-to-severe glabellar lines (frown lines) and moderate-to-severe lateral canthal lines (crow’s feet).

The agency indicted that there were issues items related to chemistry, manufacturing, and controls (CMC) processes and was not related to safety and efficacy. Galderma officials said in a press release they have already identified changes to the manufacturing process and will also be seeking further guidance from the FDA.

RelabotulinumtoxinA is ready-to-use liquid botulinum toxin A with a proprietary strain. Company officials have said a liquid formulation would eliminate the variability associated with a product from reconstituted powder.

FDA Assigns Review Date for Gene Therapy for Rare Immune Disorder

The FDA has granted priority review for Rocket Pharmaceuticals’ biologics license application (BLA) for marnetegragene autotemcel (RP-L201) to treat the rare, autosomal recessive pediatric disease leukocyte adhesion deficiency-I (LAD-I). Regulators set a Prescription Drug User Fee Act (PDUFA) date set of March 31, 2024.

Severe leukocyte adhesion deficiency-I is a caused by mutations in the ITGB2 gene that encodes for CD18, a key protein that facilitates the immune response against infections. As a result, white blood cells (leukocytes) do not function normally. Children with this disease experience life-threatening bacterial and fungal infections that respond poorly to antibiotics.

Marnetegragene autotemcel is a lentiviral vector-based investigational gene therapy. It contains autologous hematopoietic stem cells that have been genetically modified to deliver a functional copy of the ITGB2 gene. Data from the global phase 1/2 study of marnetegragene autotemcel demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 12 to 24 months of available follow-up.

Coherus Resubmits BLA for Udenyca OnBody for FDA Review

Coherus BioSciences resubmitted to the FDA the supplemental biologics license application (BLA) Udenyca OnBody, the company’s on-body injector of Udenyca (pegfilgrastim-cbqv).

Last month the FDA had issued a complete response letter due to an ongoing review of inspection findings at a third-party filler. Coherus officials said the issues have been resolved, and regulators had no issues related to clinical efficacy or safety, trial design, labeling, or device design or manufacturing.

Udenyca, a biosimilar of Amgen’s Neulasta, is used prevent infection after the use of chemotherapy for patients with nonmyeloid malignancies. Udenyca is currently approved with two product offerings: a prefilled syringe and autoinjector. Both products have a list price of $4,175, which Coherus BioScience says represents a 35% discount off Neulasta. Coherus BioSciences offers a copay program for $0 for up to an annual maximum of $15,000.

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