RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > 2021 > 12 > FDA Approvals Roundup: Apretude, Vyvgart, Tezspire

FDA Approvals Roundup: Apretude, Vyvgart, Tezspire

Posted 22 December 2021 | By Renee Matthews 

FDA Approvals Roundup: Apretude, Vyvgart, Tezspire

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

New approvals 

Apretude cleared for HIV pre-exposure prevention 

Viiv’s Apretude (cabotegravir extended-release injectable suspension) has been approved for use in at-risk adults and adolescents for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV. 

Approval of Apretude was based on safety and efficacy findings from two randomized, double-blind trials that compared Apretude against Gilead’s Truvada (emtricitabine and tenofovir disoproxil fumarate), a once daily oral medication for HIV PrEP. Trial 1 included 4,566 HIV-uninfected cisgender men and transgender women who have sex with men and have high-risk behavior for HIV infection; Trial 2 included 3,224 uninfected cisgender women at risk of acquiring HIV. Both trials measured the rate of HIV infections in the Apretude and Truvada groups. Trial 1 findings showed that participants receiving Apretude had 69% less risk of getting infected with HIV compared with those receiving Truvada. Trial 2 findings showed those taking Apretude had 90% less risk of getting infected with HIV compared with those taking Truvada. 

The HIV-1 integrase strand transfer inhibitor comes with a boxed warning for risk of drug resistance. It should be prescribed only to individuals who have tested negative for HIV immediately before the drug is administered. 

Apretude was granted priority review and breakthrough therapy designations.  

Vyvgart okayed for generalized myasthenia gravis 

argenx BV’s Vyvgart (efgartigimod) has been approved for the treatment of generalized myasthenia gravis (gMG) in adults testing positive for the anti-acetylcholine receptor (AChR) antibody. Vyvgart is a neonatal Fc receptor blocker and is the first approval in this new class of medication.  

The approval was based on findings from a 26-week clinical study of 167 patients with gMG randomized to receive Vyvgart or placebo. The findings demonstrated that 68% of patients with myasthenia gravis with anti-AChR antibodies who received Vyvgart responded to treatment during the first cycle, compared with 30% of those receiving placebo, based on an assessment of gMG’s impact on daily function. More patients receiving Vyvgart also responded to treatment on a measure of muscle weakness compared with those receiving placebo.  

The application was granted fast track and orphan drug designations.  

Tezspire gets the go-ahead for severe asthma  

Amgen’s Tezspire (tezepelumab-ekko) has been approved as an add-on maintenance treatment for adults and children aged 12 years or older with severe asthma. The biologic is a first-in-class treatment for severe asthma.  

The approval was based on findings from two randomized, double-blind, parallel group, placebo-controlled clinical trials, PATHWAY and NAVIGATOR, enrolling a total of 1,609 patients aged 12 years or older. The primary endpoint for both trials was the rate of clinically significant asthma exacerbations over 52 weeks. The annualized asthma exacerbation rate in the PATHWAY trial was 0.20 in the Tezspire group and 0.72 for placebo, and 0.93 and 2.10, respectively, in the NAVIGATOR trial. Annualized rates of exacerbations requiring emergency room visit/hospitalization were 0.03 and 0.18, respectively, in PATHWAY; and 0.06 and 0.28 in NAVIGATOR. 

Tezspire was approved following a priority review. 

New indication 

Orencia handed new indication for prevention of acute graft-versus-host disease  

Bristol Myers Squibb’s Orencia (abatacept), in combination with certain immunosuppressants, has been granted a new indication for preventing acute graft-versus-host disease (aGVHD) in adults and children aged 2 years or older undergoing stem cell transplantation from an unrelated donor. 

This is the first approval for aGVHD prevention and incorporates real-world evidence (RWE) as a component of the determination of clinical effectiveness.  

Approval of Orencia was based on findings from the double-blind, placebo-controlled GVHD-1 trial and  

GVHD-2, a registry-based clinical study conducted using real-world data (RWD) from the Center for International Blood and Marrow Transplant Research.  

In GVHD-1, 186 patients from the indicated population were randomized to receive Orencia or placebo, in combination with immunosuppressive drugs. At six months after transplantation, severe aGVHD‒free survival did not significantly improve in patients receiving Orencia compared with those receiving placebo (87% and 75%, respectively). However, overall survival rate was 97% in the Orencia group, compared with 84% in placebo patients, and moderate-severe aGVHD‒free survival was 50% and 32%, respectively. 

GVHD-2 provided additional evidence of effectiveness drawn from RWD on 54 patients from the indicated population who received Orencia plus immunosuppressive drugs and 162 patients who received standard immunosuppressive drugs alone. Six months after transplantation, overall survival was 98% among Orencia patients and 75% among those receiving immunosuppression alone. 

Orencia received breakthrough, orphan drug, and priority review designations for this indication. The FDA’s Orphan Products Grants Program provided partial support for development of the drug.  

The review was conducted under Project Orbis in collaboration with Health Canada, Swissmedic, and Israel's Ministry of Health. 

Orencia, a selective T cell costimulation modulator, was originally approved in 2005 for treating adult rheumatoid arthritis and has received subsequent approval for treating polyarticular juvenile idiopathic arthritis and adult psoriatic arthritis. 

 
 

 

© 2022 Regulatory Affairs Professionals Society.

Tags: FDA, US