Breakthroughs Blog: FDA

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. A six-month oral treatment regimen for rifampicin-resistant tuberculosis (TB) is substantially more effective than the current standard of care, according to preliminary trial results presented at the 52nd Union World Conference on Lung Health on Wednesday. The TB-Practecal trial tested a six-month regimen of bedaquiline, pretomanid, linezolid, and moxifloxacin (BPaLM) among 552 patients against the locally accepted standard of care at seven trial sites across Belarus, South Africa, and Uzbekistan. Findings showed around 89 percent of patients in the BPaLM group were cured, compared to 52 percent in the standard of care group. Current standard care for rifampicin-resistant TB can require up to 20 pills daily, along with injections, for up to 20 months.A new fingerstick blood test could be a promising tool for screening TB patients, interim study results indicate. The test, developed by Cepheid, gives results in under one hour using a few drops of blood. The new results, from a study of 195 participants in Gambia, Uganda, South Africa, and Vietnam, show an overall sensitivity of 91 percent and specificity of 86 percent, regardless of HIV status or geographical location, which meets the World Health Organization’s minimum specifications for a TB triage test. Researchers plan to expand trials to further test the technology’s accuracy, including among people who have other risk factors for TB, as well for people who have both TB and COVID-19.On Friday, the US Food and Drug Administration (FDA) released a briefing document indicating the Pfizer-BioNTech COVID-19 vaccine is more than 90 percent effective against symptomatic COVID-19 among children ages 5 to 11. The data is based on a 10-microgram vaccine dose, a third of the dose used for adults and older children, which minimizes side effects but still induces a strong immune response. The antibody response was comparable to the one seen in individuals 16 to 25 years old, and no new safety problems were identified in the companies' studies. The FDA also released an analysis later Friday showing the protection “would clearly outweigh” the risks of the shot. FDA advisers will meet Tuesday to discuss expanding authorization of the vaccine, currently greenlit for people over 12, to those ages 5 to 11.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. A US Food and Drug Administration (FDA) advisory panel voted late last week to recommend booster shots of the Moderna and Johnson & Johnson (J&J) COVID-19 vaccines. The committee voted unanimously Thursday in favor of the FDA granting emergency authorization of a half-dose booster of Moderna’s vaccine, at least six months after the second dose, among people 65 and older, those 18 to 64 with risk factors for severe COVID-19, and those whose jobs put them at high risk for serious complications of COVID-19. The following day, the same panel also recommended a second dose of the J&J vaccine given at least two months after the first shot for individuals 18 years and older. Final FDA authorization rulings on boosters are expected in the coming days.The COVID-19 pandemic has reversed years of progress against tuberculosis (TB), according to the World Health Organization’s (WHO) 2021 Global TB Report, released last week. The report, which includes data from more than 200 countries, reveals that global TB deaths increased in 2020, for the first time in more than a decade, and the number of people newly diagnosed with TB and reported to national governments fell from 7.1 million in 2019 to 5.8 million in 2020. WHO estimates an additional 4.1 million people infected with TB were not diagnosed or reported. The increase in deaths and decline in diagnoses is attributable to reduced capacity of countries to provide TB services, including diagnosis and treatment, according to WHO. The report also highlights the inadequate global funding for TB diagnostic, treatment, and prevention services, which fell from $5.8 billion in 2019 to $5.3 billion in 2020.On Wednesday, WHO announced a new team, the Scientific Advisory Group on the Origins of Novel Pathogens (SAGO), to investigate the origins of the COVID-19 pandemic, study origins of future outbreaks and epidemics, and guide studies of emerging pathogens more generally. The proposed list of SAGO team members includes 26 researchers, from 26 countries, six of whom were part of the international team that traveled to China earlier this year to study the pandemic’s origins. SAGO will conduct an independent evaluation of the available evidence on the pandemic’s origins and advise WHO about the next series of COVID-19 origin studies, as well as, beyond the current pandemic, advise WHO on the development of a global framework to define and guide studies into the origins of emerging and re-emerging pathogens of epidemic and pandemic potential.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. Merck plans to seek authorization for its COVID-19 antiviral pill after clinical trial results showed the drug, molnupiravir, cut the risk of hospitalization or death in half among high-risk individuals early in their infections. An independent board of experts monitoring the study data had recommended that the trial be stopped early because the drug’s benefit to patients had proved so convincing, and the US Food and Drug Administration agreed, according to Merck. The drug, designed to stop the virus from replicating, is taken as four capsules twice a day for five days. As an oral antiviral treatment, the pill could be a crucial new tool in combating COVID-19 by reaching more people than the antibody treatments being widely used in the United States.COVID Moonshot, an open-source effort to design a COVID-19 pill, has won US$11 million, or GBP£8 million, from the Wellcome Trust to develop a low-cost, broadly available antiviral to fight the pandemic. The project, coordinated by the Drugs for Neglected Diseases initiative, is a nonprofit consortium of scientists that began in March 2020 and led to around 250 people submitting more than 4,500 potential molecular designs intended to block the key protein that helps the virus replicate. The new funding, through Wellcome on behalf of the COVID-19 Therapeutics Accelerator, will help pay for the expensive last step of research needed to bring the project into human clinical trials. Moonshot hopes to have a compound able to be tested in humans by next year.Sanofi’s mRNA-based COVID-19 vaccine showed positive results in a phase 1/2 clinical trial, the company announced last week, providing confidence in its mRNA program that will now be used to pursue vaccines against other pathogens, including influenza. Sanofi will not be continuing to develop its mRNA COVID-19 vaccine, as the market will have been saturated by the time it would be authorized, the company said. In the preliminary phase 1/2 trial results, 91 percent of people who received the lowest tested dose saw their neutralizing antibody levels increase four-fold, a measure thought to equate to a protective level of antibodies, confirming that the mRNA platform is potent, can generate a strong immune response, and has no safety signal. Sanofi will now turn to accelerating its work on a vaccines “center of excellence” intended to be ready to produce vaccines when the next pandemic hits.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. On Thursday, US Centers for Disease Control and Prevention (CDC) Director Rochelle Walensky signed off on a recommendation from a CDC advisory panel endorsing Pfizer-BioNTech COVID-19 booster shots for millions of older or otherwise at-risk Americans, following an emergency use authorization from the US Food and Drug Administration the previous day. Dr. Walensky expanded on the advisory panel’s recommendations—which supported the use of boosters in people 65 and older, nursing home residents, and those ages 50 to 64 who have risky underlying conditions—to also include those ages 18 to 64 who are health care workers or live in institutional settings that put them at increased risk for COVID-19 exposure. Boosters will begin rollout among individuals at least six months past their last dose of the Pfizer-BioNTech vaccine.The World Health Organization (WHO) updated its COVID-19 therapeutics guidelines Friday to include a recommendation for use of Regeneron’s antibody cocktail among non-severe patients at higher risk for hospitalization. The antibody treatment, a combination of casirivimab and imdevimab, is currently recommended in the United States for use in patients with mild-to-moderate COVID-19 cases to prevent a need for hospitalization, but WHO will now work to expand its use to low- and middle-income countries where access has been more difficult due to its high cost and limited availability. WHO and Unitaid are working with manufacturers to ensure equitable distribution around the world, as well as potential donation and distribution of the drug through UNICEF.A COVID-19 vaccine candidate developed by Clover Biopharmaceuticals showed 67 percent efficacy against illness in a global phase 2/3 clinical trial, the company announced last week. The vaccine demonstrated 84 percent efficacy against moderate-to-severe COVID-19 and 100 percent efficacy against severe COVID-19 and hospitalization. The results also varied among variants of the virus, with efficacy at 79 percent against the delta variant and 59 percent against the mu variant. Clover plans to use the data to seek conditional approval later this year in China and the European Union, as well as emergency use listing from WHO, which would clear the vaccine for use by the COVAX vaccine-sharing facility, to which Clover has agreed to provide up to 414 million doses. The shot is stable under normal refrigeration conditions for at least six months, making it well-suited for global distribution.