FDA Approvals Roundup: Vijoice, Hyftor, Yescarta

Posted on April 06, 2022 | By Renee Matthews

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).

New Approvals
Vijoice approved for PIK3CA-related proliferation spectrum

Vijoice by Novartis (alpelisib) was granted accelerated approval for the treatment of severe PIK3CA-related overgrowth spectrum (PROS) manifestations in adults and children 2 years of age or older who require systemic treatment.

This indication for Vijoice, a kinase inhibitor, was approved based on real-world response rate and duration of response results in 37 patients in the single-arm group EPIK-P1 study. Patients received study drug through an expanded access program. A total of 27% of patients had a radiological response at week 24, and of these, 60% had a response that lasted 12 months or longer.

Since the indication has been approved under Accelerated Approval, its continued approval may to depend on verification and description of clinical benefit in a confirmatory trial (or trials).

The review used the Real-Time Oncology Review Pilot and Assessment Aid. The application was granted priority review and breakthrough and orphan drug designations.

Hyftor approved as topical treatment for facial angiofibromas

Hyftor from TSC Alliance (sirolimus topical gel) been approved for the treatment of facial angiofibromas in adults and children aged 6 years or over suffering from tuberous sclerosis, a rare genetic disease. Tuberous sclerosis causes non-cancerous tumors to form on or in vital organs, including the skin. Angiofibromas are small bumps that usually appear on the central surface.

Hyftor has orphan drug status for this indication. It is manufactured by Nobelpharma America.

Approval of Hyftor, an mTOR inhibitor immunosuppressant, was based on results of a randomized, multicenter, double-blind, vehicle-controlled Phase 3 study trial in 62 the patients of the indicated population. Patients applied Hyftor or vehicle to skin with angiofibroma. After 12 weeks, they were assessed against baseline photographs for improvement in facial angiofibroma size and redness. A total of 13% of Hyftor patients were rated as “improved” from baseline (vehicle, 3% of patients) and 10% were rated as “significantly improved” (vehicle, also 3%).

Rapamune (sirolimus oral solution) was originally approved in 1999 as prophylaxis of organ rejection in patients undergoing kidney transplantation.

New indications
Yescarta wins a new indication for the second-line treatment of LBCL

Kite’s Yescarta (axicabtagene ciloleucel; IV infusion) obtained a New indication for the treatment of adults with large B-cell lymphoma (LBCL) refractory to first-line chemoimmunotherapy or relapsing within 12 months of first-line chemoimmunotherapy. Cellular gene therapy is not indicated in patients with primary central nervous system lymphoma.

Approval was based on results from Phase 3, open-label, multicenter ZUMA-7 trial in which 359 adults from the indicated population were randomized 1:1 to receive Yescarta or standard second-line treatment. Event-free survival (EFS) was significantly longer in the Yescarta arm than in the standard treatment arm. The estimated EFS rate at 18 months was 41.5% in the study arm, compared to 17.0% for patients on standard treatment; the estimated median EFS was 8.3 and 2.0 months, respectively.

The review used the assessment aid. The application has been granted priority review and breakthrough therapy and orphan drug designations.

Yescarta comes with a canned warning for cytokine release syndrome and neurological toxicities. It was originally approved in 2017 for the treatment of adults with relapsed or refractory LBCL after two or more lines of systemic treatment, including diffuse large B-cell lymphoma (DLBCL), primary mediastinal LBCL, B-cell lymphoma high-grade and DLBCL resulting from follicular lymphoma.

© 2022 Society of Regulatory Affairs Professionals.


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