FDA approves new drug for treatment-resistant forms of TB that affects the lungs. The FDA approved Pretomanid in combination with bedaquiline and linezolid for the treatment of a specific type of highly treatment-resistant tuberculosis (TB) of the lungs.
“The threat of antimicrobial-resistant infections is a key challenge we face as a public health agency,” said FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD. “The bacterium that causes tuberculosis can develop resistance to the antibiotics used to treat it. Multidrug-resistant TB and extensively drug-resistant TB are public health threats due to limited treatment options. New treatments are important to meet patient national and global health needs. That’s why, among our other efforts to address antimicrobial resistance, we’re focused on facilitating the development of safe and effective new treatments to give patients more options to fight life-threatening infections. This approval also marks the second time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, a pathway, advanced by Congress, to spur development of drugs targeting infections that lack effective therapies. We hope we continue to see more development of antibacterial drugs for treating serious or life-threatening infections in limited populations of patients with unmet medical needs.”
Pretomanid in combination with bedaquiline and linezolid is approved for treating a limited and specific population of adult patients with extensively drug resistant, treatment-intolerant or nonresponsive multidrug resistant pulmonary TB. Multidrug-resistant TB (MDR TB) and extensively drug-resistant TB (XDR TB) are difficult to treat due to resistance to available therapies. According to the WHO, in 2016, there were an estimated 490,000 new cases of MDR TB worldwide, with a smaller portion of cases of XDR TB.
The safety and effectiveness of Pretomanid, taken orally in combination with bedaquiline and linezolid, was primarily demonstrated in a study of 109 patients with extensively drug-resistant, treatment intolerant, or non-responsive multidrug-resistant pulmonary TB. Of the 107 patients who were evaluated six months after the end of therapy, 95 (89 percent) were successes, which significantly exceeded the historical success rates for treatment of XDR TB.
Pretomanid is the second drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, or LPAD pathway, established by Congress under the 21st Century Cures Act to advance development and approval of antibacterial and antifungal drugs to treat serious or life-threatening infections in a limited population of patients with unmet need. Approval under the LPAD pathway may be supported by a streamlined clinical development program. These programs may involve smaller, shorter, or fewer clinical trials. As required for drugs approved under the LPAD pathway, labeling for Pretomanid includes certain statements to convey that the drug has been shown to be safe and effective only for use in a limited population.
Pretomanid also received the FDA’s Qualified Infectious Disease Product (QIDP) designation. The QIDP designation is given to antibacterial and antifungal drug products intended to treat serious or life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act.
The FDA granted Pretomanid tablets Priority Review, under which the FDA’s goal is to take action on an application within an expedited time frame, and Orphan Drug designation.
HPV
Large percent of cancers caused by HPV could be prevented by vaccine. During 2012—2016, an average of 43,999 HPV-associated cancers were reported each year, according to a new study published in CDC’s Morbidity and Mortality Weekly Report (MMWR). Among the estimated 34,800 cancers probably caused by HPV, 92 percent are attributable to the HPV types that are included in the HPV vaccine and could be prevented if HPV vaccine recommendations were followed.
CDC researchers analyzed 2012-2016 data from the CDC’s National Program of Cancer Registries and the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) to determine the incidence of HPV-associated cancers, and to estimate the annual number of cancers attributable to the HPV types in the currently available HPV vaccine. This report marks the first time these data are available at the state-level. Key findings include:
- During 2012-2016, an estimated average of 34,800 HPV-attributable cancers were diagnosed each year.
- The most common cancers were cervical (9,700) and oropharyngeal cancer (12,600).
- The number of cancers attributable to HPV types targeted by the vaccine ranged by state from 40 in Wyoming to 3,270 in California.
- Oropharyngeal cancer was the most common cancer attributable to vaccine types in all states except Texas where cervical cancer is most common.
- In Alaska, D.C., New Mexico, and New York, the estimates of oropharyngeal and cervical cancers attributable to the types in the currently available HPV vaccine were the same.
CDC recommends that all preteens get the HPV vaccine when they are 11 or 12 years old to protect them before they are ever exposed to the virus. However, additional data released in the MMWR show little progress toward increasing HPV vaccination rates among teens ages 13—17 years. This data, collected as part of the 2018 National Immunization Survey Teen (NIS-Teen), show a four percent increase in HPV vaccination rates among teen boys and less than a one percentage point increase among teen girls. Overall, just 51 percent of all teens had received all recommended doses of the HPV vaccine, a two percent increase from 2017.
HPV vaccination rates were higher in teens whose parents reported receiving a recommendation from their child’s healthcare professional. Consistently, data shows that physicians play a key role in educating parents and are the most trusted source of information for parents of pre-teens eligible for vaccination. CDC and state health departments continue to encourage health care professionals to talk to parents and provide them with information on the benefits of vaccination to prevent cancer and save lives.
The HPV vaccine is recommended for everyone through age 26 if they did not get vaccinated when they were younger. Vaccination is not recommended for people older than 26 years. However, some adults ages 27 through 45 years who are not vaccinated for HPV may decide to be vaccinated after speaking with their doctor about their risk for new HPV infections and the possible benefits of vaccination. HPV vaccination in this age range provides less benefit, as more people have likely been exposed to HPV. In addition to HPV vaccination, cervical cancer screening is recommended for women ages 21—65.
Women ages 21—29 years can be screened with a Pap test every three years. In addition to the Pap test, a test called the HPV test looks for HPV infection. Among women ages 30 to 65 years there are three strategies:
(1) Pap test alone every three years, (2) the HPV test used with the Pap test every five years, or (3) the HPV test alone every five years. The HPV test can provide additional information when Pap test results are unclear for women ages 21 and older.
Genomics
Leaders of NIH’s All of Us Research Program recap progress and next steps. The All of Us Research Program at the National Institutes of Health (NIH) has made strong progress in its efforts to advance precision medicine, according to program leadership in the New England Journal of Medicine.
With information provided by volunteers across the U.S., All of Us is developing a robust data platform to support a wide range of health studies. The program aims to include data from 1 million or more people from diverse communities. As of July 2019, more than 230,000 people have enrolled, including 175,000 participants who have completed the core protocol. Of those, 80 percent are from groups that have been historically underrepresented in biomedical research. Participants contribute information in a variety of ways, including surveys, electronic health records (EHRs), physical measurements, blood, urine and saliva samples, and Fitbit devices. In the future, the program will add new surveys and linkages to other data sets and digital health technologies and begin genotyping and whole-genome sequencing participants’ biological samples. Data will be broadly accessible to approved researchers, and participants will receive information back about themselves.
In May 2019, with enrollment ongoing, the program released initial summary data at https://www.researchallofus.org. Now, the All of Us team is planning demonstration projects to assess the usefulness and validity of the data set, in preparation for the launch of the Researcher Workbench—the secure platform where researchers will be able to conduct analyses.
The program’s ongoing success will rely on several factors. The program must continue to enroll participants from across the country, including those in rural and other underserved areas. The program needs to ensure that participants, once enrolled, derive value, remain engaged, and retain trust in the program such that they continue to share data long term. Additionally, the program must continue to protect from cyberattacks, protect participant privacy, and harmonize data from different EHR systems.
The authors anticipate that the program’s value will become even more rich as it matures, enabling new discoveries over time. A goal of the study is to improve population health through the identification of risk factors and biomarkers (including environmental exposures, habits, and social determinants) to allow more efficient and accurate diagnosis and screening, better understanding of diverse populations, more rational use of existing therapeutics, and the development of new treatments.
Reagents
MedTest Dx launches Certified Performance Guarantee Program. MedTest Dx, a solution provider offering integrated products and services for clinical diagnostic testing announced it will launch a new Certified Performance Guarantee Program for its Pointe Scientific branded line of clinical chemistry diagnostic reagents.
Pointe Scientific manufactures diagnostic reagents in the U.S., marketing its products globally under its own brand. The new Certified Performance Guarantee program is intended to provide an additional layer of assurance to customers that the company stands fully behind the quality and reliability of every Pointe Scientific branded clinical chemistry reagent it ships, issuing a free replacement to any customer who is not fully satisfied with the performance of their Pointe Scientific branded reagent.
“We are very proud of Pointe Scientific’s well-established reputation as a quality leader in diagnostic reagents, and that its products are manufactured here in the U.S.,” said Wayne Brinster, CEO of MedTest Dx. “Pointe Scientific offers an extensive line of cost-effective clinical chemistry reagents, some of which it was the first to introduce to the market. The company’s long record of successful innovation and consistent high quality made the decision to implement the Certified Performance Guarantee Program an easy one. We view the Guarantee program as indicative of our ongoing commitment to serving as our customers’ partner of choice for clinical diagnostic testing technologies.”
