FDA Approvals Roundup: Vijoice, Hyftor, Yescarta

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
 
New approvals
Vijoice okayed for PIK3CA-related overgrowth spectrum
Novartis’s Vijoice (alpelisib) has been granted accelerated approval for treating severe manifestations of PIK3CA-related overgrowth spectrum (PROS) in adults and children aged 2 years or older who require systemic therapy.
 
This indication for Vijoice, a kinase inhibitor, was approved based on real-world response rate and duration of response findings in 37 patients in the single-arm EPIK-P1 study. The patients received the study drug as part of an expanded access program. In all, 27% of patients had a radiological response at week 24, and of those, 60% had a response that lasted 12 months or longer. 
 
Because the indication was approved under the accelerated approval, its continued approval may depend on verification and description of clinical benefit in a confirmatory trial (or trials).
 
The review used the real-time oncology review pilot program and the assessment aid. The application was granted priority review and breakthrough and orphan drug designations.
 
Hyftor approved as topical treatment for facial angiofibromas
TSC Alliance’s Hyftor (sirolimus topical gel) has been approved for treating facial angiofibromas in adults and children aged 6 years or older with tuberous sclerosis complex, a rare genetic disease. Tuberous sclerosis causes noncancerous tumors to form on or in vital organs, including the skin. Angiofibromas are small bumps that usually occur on the central face.
 
Hyftor has orphan drug status for this indication. It is manufactured by Nobelpharma America.
 
Approval of Hyftor, an mTOR inhibitor immunosuppressant, was based on findings from a Phase 3 randomized, multicenter, double-blind, vehicle-controlled trial in 62 patients from the indicated population. Patients applied Hyftor or the vehicle to skin affected with angiofibroma. After 12 weeks, they were assessed against baseline photographs for improvement in size and redness of facial angiofibroma. In all, 13% of Hyftor patients were assessed as “improved” compared with baseline (vehicle, 3% of patients), and 10% were assessed as “markedly improved” (vehicle, also 3%).
 
Rapamune (sirolimus oral solution) was originally approved in 1999 as a prophylaxis of organ rejection in patients who undergo renal transplants.
 
New indications
Yescarta nabs new indication for second-line treatment of LBCL
Kite’s Yescarta (axicabtagene ciloleucel; IV infusion) has been granted a new indication for treating adults with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or relapses within 12 months of first-line chemoimmunotherapy. The cell-based gene therapy is not indicated for use in patients with primary central nervous system lymphoma.
 
Approval was based on findings from the Phase 3, open-label, multicenter ZUMA-7 trial in which 359 adults from the indicated population were randomized 1:1 to receive Yescarta or second-line standard therapy. Event-free survival (EFS) was significantly longer in the Yescarta arm than in the standard therapy arm. The estimated 18-month EFS rate was 41.5% in the study arm, compared with 17.0% for standard therapy patients; the estimated median EFS was 8.3 and 2.0 months, respectively.
 
The review used the assessment aid. The application was granted priority review and breakthrough therapy and orphan drug designations.
 
Yescarta comes with a boxed warning for cytokine release syndrome and neurologic toxicities. It was originally approved in 2017 for treating adults with relapsed or refractory LBCL after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL), primary mediastinal LBCL, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.