Biologics

FDA Accelerated Approval for Replimune Melanoma Therapy

FDA has granted accelerated approval to Replimune for its Tudriqev (vusolimogene oderparepvec-wtpg), a genetically modified oncolytic viral therapy, in combination with Bristol Myers Squibb's Opdivo (nivolumab), for treating adult patients with unresectable advanced cutaneous melanoma who experienced disease progression on a programmed death receptor-1 (PD-1)-blocking antibody-based regimen.

The regulatory green light comes on the heels of last week’s Cellular, Tissue, and Gene Therapies Advisory Committee meeting that voted 10 to 3 to recommend therapy’s approval. In briefing documents (see earlier story) released before the panel meeting, FDA reviewers had argued that the company’s BLA relied on a single-arm trial without an adequate control group, making it impossible to determine the contribution of Tudriqev to the responses observed with the combination regimen. Agency staff also questioned aspects of the trial's response assessment, saying they may have artificially inflated efficacy results.

During the panel meeting, committee members acknowledged the limitations of the data package but also cited the lack of effective treatment options for patients with anti-PD-1 refractory melanoma. Supporters of the therapy argued that the durable responses observed in heavily pretreated patients and the unmet medical need justified approval despite the uncertainties surrounding the trial design.

The approval is the latest turn in a regulatory saga that has stretched nearly two years and two previous rejections. The June BLA resubmission followed FDA's April complete response letter that rejected RP1 despite clinical trial results showing tumor shrinkage or disappearance in roughly one-third of patients. The decision, together with several other high-profile regulatory setbacks issued this spring, drew criticism from industry groups and investors, who argued the agency had become less predictable under former FDA commissioner Marty Makary and the Trump administration. (see earlier story).

RP1 is a genetically engineered virus designed to destroy tumor cells and stimulate immune activity alongside the immunotherapy drug Opdivo. An FDA release says the “safety and efficacy of Tudriqev were evaluated in IGNYTE (NCT03767348), an open-label, multiregional, single-arm trial in 140 adult patients with Stage IIIB, IIIC or IV unresectable advanced melanoma who experienced disease progression on at least eight consecutive weeks of prior anti-PD-1-based therapy. The major efficacy outcome measures were objective response rate (24.2%) and duration of response (14.1 months).

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

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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