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. An experimental drug, remdesivir, was shown to protect monkeys against infection with Nipah virus—an emerging pandemic threat for which there is no approved vaccine or cure. The only current treatment is a still experimental monoclonal antibody that was tested during an outbreak in India last year. Remdesivir is also being tested against the Ebola virus in the current outbreak in the Democratic Republic of Congo and has shown some effectiveness against Lassa fever, MERS coronavirus, and respiratory syncytial virus in other studies. In the new study performed by the National Institute of Allergy and Infectious Diseases (NIAID) and the Centers for Disease Control and Prevention, eight African green monkeys were given lethal doses of Nipah virus. The four monkeys who received intravenous remdesivir within 24 hours of infection and daily infusions for 12 days survived, while the four that did not died within eight days. If remdesivir wins approval, “it will give us an extra treatment that could be used relatively quickly,” said Emmie de Wit, a virologist at NIAID, noting that “the average person who reaches a hospital dies within two days, so it’s hard to protect them once they’re infected.” The next step is to test how long after infection the drug can be given and still cure the animals. Women make up just over half of the 35 million people living with HIV worldwide but are underrepresented in clinical trials of potential HIV treatments, cures, and vaccines. A 2016 study performed by amfAR, the Foundation for AIDS Research, found that women represented a mean of only 11 percent of HIV cure trials, 19 percent of antiretroviral drug trials, and 38 percent of vaccine studies. Despite the well-known differences in how the immune systems of men and women respond to the HIV infection and how women respond to some drug treatments, clinical trials continue to rely heavily on the participation of gay men in particular. Further complicating women’s participation in clinical trials is the US Food and Drug Administration’s (FDA) strict rules for including women of childbearing age, leaving most researchers to opt for the easy way out and just enroll men, collecting data from women only after the drug is on the market. As scientists continue to pursue cures for HIV in light of reports of the second patient cured of the infection, researchers must reckon with the longstanding obstacle of the lack of women in clinical trials. The FDA has authorized the first diagnostic test to detect Zika antibodies in human blood to be marketed in the United States. The diagnostic, known as ZIKV Detect 2.0 IgM Capture ELISA, detects the proteins produced by the body’s immune system when it tests for the Zika virus in blood. Though 18 other active tests to detect Zika exist, they have only been allowed under emergency use. This newly approved diagnostic test will only be usable by those with clinical signs and symptoms of Zika or on those who have traveled to regions with active Zika transmissions. FDA Acting Commissioner Ned Sharples noted that the marketing authorization of this Zika diagnostic demonstrates “FDA’s work to protect the public health in emergency response situations.”

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 report released by the Economist Intelligence Unit warns that the fight against drug-resistant tuberculosis (DR-TB) lags behind that of other diseases in terms of money and public attention. Deaths from DR-TB will cost the world economy nearly US$20 billion annually; however, international funding for TB is approximately one-eighth that of HIV and AIDS and about half that of malaria. The report calls for TB to be treated as a public health emergency and states that $9 billion is needed by 2020 to develop new drugs, diagnostics, and vaccines to address this disease—the world’s number one infectious disease killer. DR-TB can develop when patients fail to adhere to the notoriously grueling treatment regimen, which includes thousands of pills and painful injections, or because of poor-quality drugs. Last year, the United Nations held the first-ever High-Level Meeting on TB, determining that ending TB is a global priority after progress against the disease has stalled in recent years. The World Health Organization (WHO) announced plans last Tuesday to change its vaccination strategy in the Democratic Republic of Congo (DRC) in an attempt to better confront the country’s current Ebola outbreak, which has lasted nine months and claimed more than 1,000 lives. As part of the new vaccination strategy, WHO intends to launch “pop-up” vaccination campaigns in safe communities and shift to immunizing as many people in each target area as possible instead of relying solely on its current tactic of ring vaccinating health workers and the immediate contacts of each known case. To extend vaccine supplies, WHO also recommended offering smaller doses of the vaccine—developed by Merck—to those at high risk and low risk respectively, and eventually introducing a second vaccine made by Johnson and Johnson. A consortium, including the Coalition for Epidemic Preparedness Innovations and the London School of Hygiene and Tropical Medicine, is planning how to safely roll out the new vaccine. On Wednesday, the US Food and Drug Administration (FDA) released two new draft documents which provide guidance about how to study drug safety and efficacy in pregnant and lactating women. The draft documents provide insight to drug makers into the FDA’s thinking on when they should study a medicine in lactating women and how they can better monitor health outcomes in pregnant women who are taking medications that have already been FDA-approved. These documents follow 2018 recommendations made by a federal task force formed by Congress to study the lack of research involving pregnant and lactating women. Historically, the drug industry has excluded pregnant women from research out of concern that an experimental medication might harm a fetus. There is a similarly small amount of research on medications safety in lactating women. Once finalized, the documents will help companies collect more high-quality safety information about drugs used in pregnant and lactating women.

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 landmark study found that men whose HIV infection was fully suppressed by antiretroviral drugs had no chance of infecting their partner—suggesting that if everyone with HIV were fully treated, there would be no further infections. Of the nearly 1,000 male couples across Europe who participated in the study, in which one partner with HIV was receiving treatment to suppress the virus, there were no cases of transmission of the infection to the HIV-negative partner during sex without a condom. According to Professor Alison Rodger, co-leader of the paper published in The Lancet, these findings “provide conclusive evidence for gay men that the risk of HIV transmission with suppressive ART [antiretroviral therapy] is zero” and that the study’s findings “support the message of the international U=U campaign that an undetectable viral load makes HIV untransmittable.” The Global Polio Eradication Initiative (GPEI) unveiled a new five-year strategy on Tuesday, which includes the use of novel oral polio vaccines—several of which are being developed by PATH and its partners—as well as the adoption of smartphone technologies to assist in disease surveillance and faster response. GPEI is also planning to establish a regional hub in Amman, Jordan to allow for easier coordination and increased technical support for polio vaccination programs in Afghanistan and Pakistan, two polio-endemic countries. Additionally, as part of the new strategy, GPEI is expanding its partnership with development institutions such as Gavi, the Vaccine Alliance. These new approaches, among others, will be implemented by GPEI under its new strategy from 2019-2023 in the hopes that this new strategy will be its last, with wild poliovirus successfully eliminated by 2023. The new strategy is currently facing a funding gap of US$3.27 billion, which organizers hope to fill with a pledging conference in November. On Wednesday, the US Food and Drug Administration (FDA) approved the first vaccine against dengue fever. The vaccine, Dengvaxia, has generated much controversy in recent months due to post-marketing research performed by its manufacturer, Sanofi Pasteur, which showed that the vaccine offers protection for people who have already had at least one bout of dengue, but increases the risk of severe infection in children who were dengue-naïve, or never infected when they were vaccinated. The FDA ruled that Dengvaxia can only be administered to individuals between the ages of 9-16 living in parts of the United States where the dengue virus is endemic, and that it can only be given to children and young adults who had one previous laboratory-confirmed case of dengue. Identifying these individuals with prior infection is currently a challenge, because there is no point-of-care diagnostic test available, though Sanofi officials say they have to have one ready for FDA submission by late 2020. Sanofi will earn a priority review voucher (PRV) by bringing Dengvaxia to market. A PRV is an incentive created to encourage pharmaceutical companies to develop drugs and vaccines for neglected diseases and, in exchange, allows a company to expedite the review of another product it wants to bring to market or sell the voucher to another company.

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 phase II clinical trial, led by the Drugs for Neglected Diseases initiative (DNDi), found that a two-week treatment of benznidazole was just as effective in treating Chagas— a parasitic disease which causes permanent damage to the heart as well as the nervous and digestive systems—as the conventional eight-week treatment course. The shorter treatment means reduced side effects, which range from skin rashes to peripheral neuropathy, and thus also improved treatment adherence—both of which are critical for the control and elimination of Chagas disease. DNDi hopes to make the disease less debilitating for future patients “and turn this breakthrough discovery into a reality for people affected by the disease.” On Monday, March 11, the Trump administration released its budget proposal for fiscal year 2020. While the budget proposal requested US$291 million for the President’s domestically-focused “Ending the HIV epidemic” plan, —which includes funding for biomedical interventions—the global health community has called out the proposed reduction in funding to fight HIV globally also included in the President’s 2020 budget proposal. Overall, the President’s Budget Request proposes deep cuts to biomedical research across a wide range of federal agencies and to global health programs at the Department of State and the US Agency for International Development. TB Alliance announced that the US Food and Drug Administration (FDA) has accepted a new drug application for pretomanid, a novel tuberculosis (TB) drug candidate, as part of a treatment regimen for extensively drug-resistant TB; treatment-intolerant, multidrug-resistant TB; and treatment-nonresponsive, multidrug-resistant TB. Pretomanid would be used in combination with bedaquiline and linezolid. In a clinical trial in South Africa using this treatment regimen, 89 percent of study participants resolved their clinical infection after six months of treatment and six months of post-treatment follow up. The FDA granted priority review of this new drug application leveraging the Prescription Drug User Fee Act with an action date expected in the third quarter of this year.