OPIOID EPIDEMIC
FDA offers resources to address opioid misuse
The FDA has made reducing the number of Americans addicted to opioids and cutting the rate of new addiction one of its highest priorities. Strategies include ensuring that only appropriately indicated patients are prescribed opioids and that the prescriptions are for durations and doses that properly match the clinical reason for which the drug is being prescribed. Other initiatives include:
* supporting treatment of those with opioid use disorder
* fostering development of novel pain management therapies
* improving enforcement of laws and regulations that prohibit the sale of illicit drugs online and through the mail.
To support each of these goals, the FDA offers a collection of links to resources such as information about medication-assisted treatment, proper disposal of unwanted medications, abuse-deterrent opioid analgesics, and patient-focused development of drugs for chronic pain. Access it at http://www.fda.gov/drugs/information-drug-class/opioid-medications.
Source: Food and Drug Administration. Opioid medications. March 29, 2021. http://www.fda.gov/drugs/information-drug-class/opioid-medications.
DUCHENNE MUSCULAR DYSTROPHY
Therapy approved for rare mutation
Amondys 45 (casimersen) injection has been approved to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. Exons are pieces of DNA in a person's genome that provide information for making proteins. Approximately 8% of patients with DMD have a mutation that is amenable to exon 45 skipping.
The most common type of muscular dystrophy, DMD is characterized by progressive muscle deterioration and weakness. It is caused by genetic mutations causing an absence of dystrophin, a protein found in muscle fiber. Most patients with DMD are male and experience the first signs and symptoms between ages 3 and 5 years.
In a double-blind, placebo-controlled clinical trial, production of dystrophin in skeletal muscle increased in patients receiving the new drug. This is the first FDA-approved targeted treatment for patients with this type of mutation.
Although kidney toxicity was not observed in clinical studies, it was observed in the nonclinical studies and has also occurred after administration of some similar drugs. Closely monitor kidney function in patients taking Amondys 45.
Source: US Food and Drug Administration. FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation. News release. February 25, 2021.
GASTRIC CANCER
First immunotherapy for advanced disease
The FDA recently approved a new indication for nivolumab (Opdivo) to be used in combination with certain types of chemotherapy for the initial treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma. Nivolumab has been previously approved for many other indications, including melanoma, non-small cell lung cancer, and malignant pleural mesothelioma.
This new combination is the first FDA-approved immunotherapy for first-line treatment of gastric cancer. Nivolumab received Priority Review and Orphan Drug designations for this indication. According to an FDA spokesperson, this is the first therapy "in more than a decade to show a survival benefit for patients with advanced or metastatic gastric cancer who are being treated for the first time." With current therapies, survival for patients with gastric cancer is poor; the survival rate for all stages is just 32%.
Nivolumab is a monoclonal antibody that inhibits tumor growth by enhancing T-cell function. Its efficacy was evaluated in a randomized, multicenter, open-label trial of 1,581 patients with previously untreated advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma. Median survival was 13.8 months for patients who received nivolumab plus chemotherapy, compared with 11.6 months for patients who received chemotherapy alone.
Nivolumab can cause serious immune-mediated adverse reactions, including pneumonitis, colitis, hepatitis, endocrinopathies, and nephritis. Before starting treatment, patients should tell their healthcare provider if they have immune system, pulmonary, or liver problems; have had an organ transplant; or are pregnant or plan to become pregnant.
Approximately 28,000 new cases of gastric cancer are diagnosed each year in the US, according to the FDA.
Source: Food and Drug Administration. FDA approves first immunotherapy for initial treatment of gastric cancer. News release. April 16, 2021.
COVID-19
Universal coronavirus vaccine on the horizon?
Researchers developing an innovative vaccine against SARS-CoV-2, the virus causing COVID-19, suggest that their novel approach could provide protection against existing and future strains of the COVID-19 coronavirus as well as other coronaviruses, possibly including those that cause the common cold. Unlike other COVID-19 vaccines currently in use, this approach targets a part of the virus's spike protein that is essentially universal among coronaviruses. This protein has not been observed to differ in the many genetic sequences of SARS-CoV-2 found so far around the world during the pandemic.
The new vaccine-production process involves synthesizing DNA that directs the immune system to mount a protective immune response against the virus and inserting it into genetically engineered Escherichia coli bacteria, which serve as a vector. Because much of the bacteria's natural genetic material has been removed, the ability of the immune system to recognize and respond to the vaccine antigen it carries is greatly increased.
Many low- to middle-income countries around the world currently make vaccines using similar technology for only about $1 per dose and could easily adapt to produce the new vaccine, the researchers say. In addition, these types of vaccines can be produced in a matter of weeks, an important advantage for managing current and future pandemics.
Additional testing and human trials must be conducted before the vaccine is submitted to the FDA for review and approval.
Source: University of Virginia Health System. New COVID-19 vaccine may offer broad protection from coronaviruses. News release. April 19, 2021.