Drugs

Pfizer Optimistic on Hemphilia A Gene Therapy

Pfizer says it will discuss with FDA plans to seek approval of its investigational gene therapy for treating adults with moderately severe to severe hemophilia A. The move follows just-announced “positive topline results” from the Phase 3 AFFINE study (NCT04370054), according to the company.

“The AFFINE study achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment,” Pfizer says. After a single dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73), it says.

Regarding key secondary endpoints, these were met too by demonstrating superiority compared to prophylaxis. Pfizer says 84% of participants maintained FVIII activity greater than 5% at 15 months post-infusion with the majority having FVIII activity greater than 15%. “Throughout the study, among all dosed participants, one participant (1.3%) returned to prophylaxis post-infusion,” it adds.

On the safety side, the gene therapy was generally well tolerated, according to Pfizer. “Transient elevated FVIII levels greater than 50% were observed in 49.3% of dosed participants, as measured via chromogenic assay, with no impact on efficacy and safety results,” it says. “Serious adverse events were reported in 15 patients (20%), including 13 events reported by 10 patients (13.3%) assessed as related to treatment. Treatment-related adverse events generally resolved in response to clinical management.”

Giroctocogene fitelparvovec has been granted orphan drug, fast track and regenerative medicine advanced therapy designations from FDA.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome