ViiV Healthcare announces FDA Priority Review designation for cabotegravir long-acting for prevention of HIV

London, XX September 2021 – ViiV Healthcare, the global specialist HIV company majority owned by GSK, with Pfizer Inc. and Shionogi Limited as shareholders, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for a New Drug Application (NDA) for investigational, injectable cabotegravir long-acting for the prevention of HIV, also called pre-exposure prophylaxis, or PrEP. The FDA’s Priority Review designation is for drugs considered significant improvements in the treatment, diagnosis, or prevention of serious disease, and means the FDA’s goal is to take action on a new drug application (NDA) within six months (compared to ten months under standard review). The FDA has set a target approval date of 24 January 2022.

The NDA was based on the results from two phase IIb/III studies, HPTN 083 and HPTN 084, which evaluated the safety and efficacy of cabotegravir long-acting for PrEP in men who have sex with men, transgender women, and cisgender women.^1,2 The blinded, randomized portions of both studies were stopped early by an independent Data Safety Monitoring Board after cabotegravir was shown to be superior to daily oral emtricitabine/tenofovir disoproxil fumarate (FTC/TDF) tablets in preventing the acquisition of HIV.^1,2 If approved, cabotegravir would be the first, long-acting therapy for HIV PrEP.

The Priority Review designation of cabotegravir long-acting for PrEP builds upon its prior identification as a Breakthrough Therapy by the FDA. The FDA’s Breakthrough Therapy designation is intended to facilitate the development and expedite the review of drugs that address serious or life-threatening medical conditions. It results in closer and more frequent collaboration with the FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support its approval.
Kimberly Smith, M.D., MPH, Head of Research & Development at ViiV Healthcare, said: “The FDA’s Priority Review designation of cabotegravir long-acting for PrEP underscores the importance of this medicine, driven by the remarkable results of the HPTN studies, which demonstrated cabotegravir’s superiority over the current standard of care. In the United States, fewer than 25% of those who could benefit from PrEP are currently taking it, illustrating the need for additional HIV prevention options. We believe new, effective prevention options like investigational cabotegravir long-acting for PrEP will help play a significant role in our collective efforts to end the HIV epidemic.”

ViiV Healthcare will submit cabotegravir long-acting for PrEP to global regulatory authorities by the end of 2021. Cabotegravir long-acting for PrEP has not been approved or licensed anywhere in the world for use in HIV prevention.

About HPTN 083 (NCT02720094)

The HPTN 083 study is a phase IIb/III double blind study designed to evaluate the safety and efficacy of long-acting injectable cabotegravir for HIV prevention administered every eight weeks compared to daily oral FTC/TDF tablets (200 mg/300 mg). The trial design included an oral lead-in phase to assess tolerability to cabotegravir before administering the intramuscular injection. Each participant was to receive a maximum of three years of blinded study medication. The study opened to enrolment in November 2016. HPTN 083 was conducted in approximately 4,566 men who have sex with men and transgender women who have sex with men at research centers in Argentina, Brazil, Peru, United States, South Africa, Thailand and Vietnam.¹

Among the 51 people in the trial who acquired HIV, 12 were randomized to the long-acting cabotegravir arm and 39 were randomized to the daily, oral FTC/TDF arm. This translated to an HIV incidence rate of 0.38% (95% confidence interval [CI] 0.20%-0.66%) in the cabotegravir group and 1.22% (95% CI 0.86%-1.66%) in the FTC/TDF group. Adherence to oral FTC/TDF was high, based on a random subset sampling that detected tenofovir (> 0.31 ng/ml) in 86% of all samples tested. Despite this high level of adherence to oral therapy, long-acting cabotegravir was 69% (95% CI 41%-84%) more effective than FTC/TDF in preventing HIV acquisition in the study population.

Safety was similar in the two groups. Most participants in the cabotegravir group (80%) reported pain or tenderness at the injection site, compared to only 31% of those in the FTC/TDF arm, who received placebo injections. Discontinuation due to injection site reactions or injection intolerance in the cabotegravir arm of the study was 2% and there were no discontinuations due to ISRs in the FTC/TDF arm. For further information on HPTN 083 please see https://clinicaltrials.gov/ct2/show/NCT02720094.

About HPTN 084 (NCT03164564)

The HPTN 084 study is a phase IIb/III double blind safety and efficacy study designed to evaluate the safety and efficacy of the long-acting injectable cabotegravir for HIV prevention administered every eight weeks compared to daily oral FTC/TDF tablets (200 mg/300 mg) in 3,223 women who are at increased risk of HIV acquisition. The trial design included an oral lead-in phase to assess tolerability to cabotegravir before administering the intramuscular injection.

HPTN 084 opened to enrolment in November 2017 and is being conducted at research centers in Botswana, Kenya, Malawi, South Africa, Eswatini, Uganda and Zimbabwe.²

Among the 40 women in the trial who acquired HIV, four were randomized to the long-acting cabotegravir arm and 36 were randomized to the daily, oral FTC/TDF arm. This translated to an HIV incidence rate of 0.21% (95% confidence interval [CI] 0.06% – 0.526%) in the cabotegravir group and 1.86% (95% CI 1.3%-2.57%) in the FTC/TDF group. While both methods were highly effective at preventing HIV acquisition, long-acting cabotegravir was 89% (95% CI 68-96%) more effective than FTC/TDF.

Long-acting cabotegravir and FTC/TDF tablets were both well tolerated throughout the study, with most adverse events being mild or moderate in severity and with the frequency largely balanced between both treatment arms. Injection site reactions (ISRs) were low in both groups and represented numerical improvements from what was demonstrated in the HPTN 083 study in men. ISRs in HPTN 084 occurred more frequently in the cabotegravir arm (32%) vs. the FTC/TDF arm (9%), which received placebo injections. There were no discontinuations due to injection site reactions or injection intolerance in either arm of the study. Gastrointestinal disorders and nausea were more common in the FTC/TDF arm. For further information please see https://clinicaltrials.gov/ct2/show/NCT03164564.

About ViiV Healthcare

ViiV Healthcare is a global specialist HIV company established in November 2009 by GlaxoSmithKline (LSE: GSK) and Pfizer (NYSE: PFE) dedicated to delivering advances in treatment and care for people living with HIV and for people who are at risk of becoming infected with HIV. Shionogi joined in October 2012. The company’s aims are to take a deeper and broader interest in HIV/AIDS than any company has done before and take a new approach to deliver effective and innovative medicines for HIV treatment and prevention, as well as support communities affected by HIV.

For more information on the company, its management, portfolio, pipeline, and commitment, please visit www.viivhealthcare.com.

About GSK

GSK is a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer. For further information please visit www.gsk.com/about-us/.

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