FDA pushes back Ascendis' PDUFA for long-acting hormone therapy; Debiopharm takes home a win in mid-stage test for ADC
Danish biotech Ascendis Biopharma has been working on a treatment for pediatric growth hormone deficiency, but the FDA says they’re going to have to wait a little bit longer before they can make their approval decision.
Regulators extended the review period by three months on Monday, Ascendis said, pushing back the PDUFA date to September 25. The extension was in response to additional information Ascendis submitted as part of its BLA.
“We have responded to all outstanding questions from the FDA and believe the complete package we have submitted satisfies all of FDA’s requests,” CEO Jan Mikkelsen said in a statement.
The program in question is known as lonapegsomatropin, designed to deliver the same unmodified human growth hormone used in daily therapies, but at a predictable rate once a week. Researchers have been working on a therapy for children, as well as for adults with growth hormone deficiency.
In their Phase III pediatric trial back in 2019, the weekly therapy not only demonstrated non-inferiority to a daily version, but also showed superiority after one year. Ascendis is hoping to capitalize on those results, which surprised analysts at the time, by producing what would be the first long-acting hormone therapy. — Max Gelman
A non-Hodgkin’s lymphoma ADC shows positive response in 30% of patients
Phase II data from Debiopharm’s ADC naratuximab emtansine has shown promise for the treatment of one of the most aggressive forms of non-Hodgkin’s lymphoma, the company said Monday.
The complete response rate was 31.6% in all patients with diffuse large B cell lymphoma, and 32% in patients with more than two prior therapies. About 8% of patients stopped treatment because of side effects.
Debiopharm CEO Bertrand Ducrey said in a statement:
We now see this alternative combination targeting both CD37 and CD20 could address the unmet need of DLBCL patients who have relapsed or not responded to earlier lines of treatment. These promising efficacy results and good safety profile are motivation for further exploration of the potential benefit that this potent ADC technology could bring to patients. We’ve clearly arrived at the stage of selecting a partner to unfold the full value of this novel therapeutic option.
Naratuximab emtansine treats patients with diffuse large B-cell lymphoma, which accounts for about 30-40% of all non-Hodgkin’s lymphoma cases. About half of patients aren’t cured by the first round of treatment. Naratuximab emtansine targets the CD37 antigen to release a toxic DM1 payload. The duration of response lasted longer than one year for 66% of patients, the company said. — Josh Sullivan