Kresladi becomes the first FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2, and Awiqli the first and only once-weekly basal insulin treatment for adults with type 2 diabetes.
Two FDA approvals announced this week mark meaningful advances in diabetes management and rare pediatric disease, with Novo Nordisk and Rocket Pharmaceuticals each bringing first-of-their-kind therapies to market.
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FDA has approved Awiqli (insulin icodec-abae) injection 700 units/mL as the first and only once-weekly basal insulin for adults with type 2 diabetes.1 The drug is indicated as an adjunct to diet and exercise to improve blood sugar control and is expected to launch in the U.S. in the second half of 2026 using the FlexTouch delivery device.1
The approval follows a difficult application process, which saw Novo Nordisk
Awiqli is already approved in the EU and 13 additional countries.
The approval is based on the Onwards Phase III program, which comprised four randomized, active-controlled trials in approximately 2,680 adults with uncontrolled type 2 diabetes. The trials evaluated once-weekly Awiqli against daily basal insulin across multiple treatment combinations, including with mealtime insulin, common oral antidiabetic agents, and GLP-1 receptor agonists.
Awiqli met the primary endpoint of HbA1c reduction across the program, with an overall safety profile consistent with the daily basal insulin class.
Mike Doustdar, president and CEO of Novo Nordisk, framed the approval as a reaffirmation of the company's commitment to insulin innovation at a time when other industry players have retreated from the space.
"At a time when parts of the industry are stepping back from insulin, we are reaffirming our commitment, continuing to invest in innovation, access, and supply for the millions of patients who rely on insulin every day," he said.
In other regulatory news, FDA granted accelerated approval to Kresladi (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) who lack a matched sibling donor for conventional stem cell transplant.2
Kresladi is the first approved gene therapy for LAD-I and offers an alternative path for patients who lack a suitable donor for allogeneic transplant, historically the only curative option.
LAD-I is an ultra-rare genetic disease caused by mutations in the ITGB2 gene, which encodes the CD18 protein needed for white blood cells to adhere to blood vessel walls and migrate to tissues to fight infection.
Infants with severe LAD-I suffer from recurrent, life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. The condition affects an estimated one in 100,000 to one in 200,000 live births in the U.S., with roughly two-thirds of cases classified as severe.2
The accelerated approval is based on increases in neutrophil CD18 and CD11a surface expression, with confirmation of clinical benefit contingent on longer-term follow-up data from the ongoing clinical study and a post-marketing registry.2
In connection with the approval, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher, which the company said it intends to monetize to enhance financial flexibility.2
Gaurav Shah, CEO of Rocket Pharmaceuticals, says the approval reflects years of collaboration among patients, families, investigators, and regulators.
Donald Kohn, principal investigator of the Phase I/II study and distinguished professor at UCLA, says the approval represents the culmination of many years of scientific research aimed at addressing the underlying cause of a devastating disease.
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