FDA Roundup: Biosimilar Approval, Hereditary Angioedema Treatment Receives Orphan Drug Designation
Check out important updates from the FDA for the week of September 30.
FDA Approves Stelara Biosimilar Ustekinumab-aauz
FDA Roundup: Biosimilar Approval, Hereditary Angioedema Treatment Receives Orphan Drug Designation / Tada Images - stock.adobe.com
The FDA approved ustekinumab-aauz (Otulfi) for the treatment of Crohn’s disease, ulcerative colitis, moderate to severe plaque psoriasis and active psoriatic arthritis, Fresenius Kabi and Formycon announced in a release.1 Otulfi is a biosimilar referencing Johnson and Johnson’s ustekinumab (Stelara).
The approval of ustekinumab-aauz was based on data from a comprehensive package, including analytical, pre-clinical, clinical and manufacturing data. The data showed that ustekinumab-aauz demonstrated comparable efficacy, safety, pharmacokinetics and immunogenicity to Stelara in patients with moderate to severe plaque psoriasis. The therapy was approved for subcutaneous and intravenous formulations.
“The FDA approval of Otulfi, Fresenius Kabi’s fourth biosimilar product in the U.S. market, is an important milestone on our pathway to consistently broadening our biopharma portfolio in the US and worldwide,” Dr. Sang-Jin Pak, president of Biopharma and member of the Fresenius Kabi Management Board, said in a release.1 “In line with our Vision 2026 growth strategy, we are fully committed to becoming a significant player in the biopharma field and offering essential treatment options for patients globally.”
FDA Grants Orphan Drug Designation to Hereditary Angioedema Treatment
The FDA granted Orphan Drug Designation to navenibart (STAR-0215), a monoclonal antibody inhibitor of plasma kallikrein for the treatment of hereditary angioedema, Astria Therapeutics announced in a release.2 Orphan Drug Designation provides companies with development and commercial incentives for designated compounds and medicines.
The FDA designation was given to Astria based on data from the phase 1b/2 ALPHA-STAR clinical trial. In the study, navenibart demonstrated a favorable safety and tolerability profile, as well as a reduction of monthly attack rates by 90% to 96% when dosed once or twice over 6 months.
“Receiving orphan drug designation for navenibart is an important affirmation of our belief that there is a significant unmet need for people living with HAE,” Jill C. Milne, PhD, CEO of Astria, said in a release.2 “We believe navenibart has the potential to be the market-leading hereditary angioedema treatment because of its trusted mechanism and modality, efficacy observed to date, and low treatment burden with infrequent dosing, and think that navenibart could change the way that people live with their hereditary angioedema.”
FDA Grants Rare Pediatric Disease Designation to Congenital Muscular Dystrophy Type 1a Treatment
The FDA granted Rare Pediatric Disease Designation to MDL-101, a novel precision medicine for the treatment of congenital muscular dystrophy type 1a, Modalis Therapeutics announced in a release.3 Rare Pediatric Disease Designation is granted by the FDA to treatments for serious and life-threatening diseases that primarily affect children under the age 18 years.
MDL-101 is an experimental, epigenetic editing therapy that is comprised of a guide nucleotide, which targets the LAMA1 gene. The therapy works by upregulating LAMA1 gene products in the muscle tissue of patients to compensate for loss-of-function caused by mutation of LAMA2. It has the potential to provide a one-time, durable treatment.
“We are pleased that the FDA has recognized our development efforts for the rare disease and granted us Rare Pediatric Disease Designation,” Haru Morita, CEO of Modalis, said in a release.3 “We have received many requests for our efforts from children and families around the world suffering from this disease for which there is currently no treatment, and we feel a mission to respond to the expectations of patients who are eagerly awaiting the start of clinical trials as soon as possible.”
