Key FDA Approvals on Novel Treatment Options for Menopausal Hot Flashes, CDI, Opioid Overdoses, and More

FDA Approves Novel Treatment for Menopausal Hot Flashes¹

The FDA has approved fezolinetant as the first neurokinin 3 receptor antagonist approved for the treatment of moderate to severe vasomotor symptoms caused by menopause. 

Vasomotor symptoms, often called hot flashes, occur in approximately 80% of individuals experiencing menopause. Many women who experience vasomotor symptoms have a history of bleeding, stroke, or other comorbidities that prevent them for taking hormone therapies to manage these symptoms. Fezolinetant works by binding to and blocking the activities of the neurokinin 3 receptor, which targets the neural activity causing vasomotor symptoms without the need for hormones.

The approval was based on the first 12-weeks of two randomized, double-blind phase 3 trials, which first examined the effectiveness of fezolinetant. After 12 weeks, individuals initially assigned to placebo were then re-randomized to receive treatment for a 40-week extension study to examine the safety of fezolinetant. Both trials ran for a total of 52 weeks.

Read the full FDA alert here.

First Oral Treatment to Prevent Recurring Clostridioides difficile Infection Approved²

The FDA has approved the first fecal microbiota product for the prevention of recurring Clostridioides difficile (C. difficile) infection (CDI). The oral treatment is approved for individuals aged 18 years and older, following antibacterial treatment for recurrent CDI.

CDI is one of the most common healthcare-associated infections in the United States that is caused by the bacterium C. difficile. According to the FDA’s press release, the infection causes 15,000 to 30,000 deaths annually.

The approval follows a randomized, double-blind, placebo-controlled clinical study and an open-label clinical study—both tested on the safety of the treatment. Across both studies, participants (n = 346) had recurrent CDI, were 48 to 96 hours post-antibacterial treatment, and their symptoms were controlled. In an analysis among 90 oral treatment recipients, when compared to 92 placebo recipients, the most common adverse events were abdominal bloating, fatigue, constipation, chills, and diarrhea.

Read the full FDA alert here.

FDA Approves First Prescription Nasal Spray to Reverse Opioid Overdose³

The FDA has approved the first nalmefene hydrochloride nasal spray for the emergency treatment of known or suspected opioid overdose. The treatment’s use is approved for adults and pediatric patients aged 12 years and older.

Nalmefene—an opioid receptor antagonist—is typically used to treat acute opioid overdose. If administered quickly, the treatment can reverse the effects of opioid overdose (respiratory depression, sedation, low blood pressure). This is the FDA’s first approval of nalmefene hydrochloride nasal spray for health care and community use.

The approval of the nalmefene hydrochloride treatment follows safety and pharmacokinetic studies, and a study that assessed how quickly the drug worked in people who use opioids recreationally.

Read the full FDA alert here.

First Oral Treatment Approved to Treat Moderate, Severe Crohn Disease⁴

The FDA has approved upadacitinib to treat adults with moderate-to-severe Crohn disease. Upadacitinib is the first oral treatment approved by the FDA to treat this disease. The FDA has previously approved upadacitinib for several other conditions including moderate-to-severe rheumatoid arthritis, psoriatic arthritis, and atopic dermatitis.

The approval follows two randomized induction trials that evaluated the efficacy and safety of the oral treatment. In total, 857 patients with moderately to severely active Crohn disease were randomized 2:1 and received 45 mg of treatment or placebo once a day for 12 weeks. At the last week of treatment, a greater proportion of patients who received upadacitinib achieved clinical remission compared to those who received placebo.

Read the full FDA alert here.

References

1. FDA approves novel drug to treat moderate to severe hot flashes caused by menopause. News release. US Food and Drug Administration; May 12, 2023. Accessed June 5, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-novel-drug-treat-moderate-severe-hot-flashes-caused-menopause.
2. FDA approves first orally administered fecal microbiota product for the prevention of recurrence of Clostridioides difficile Infection. News release. US Food and Drug Administration; April 26, 2023. Accessed May 31, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-orally-administered-fecal-microbiota-product-prevention-recurrence-clostridioides.
3. FDA approves first oral treatment for moderately to severely active Crohn disease. News release. US Food and Drug Administration; May 18, 2023. Accessed May 25, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-moderately-severely-active-crohns-disease.
4. FDA approves first oral treatment for moderately to severely active Crohn disease. News release. US Food and Drug Administration; May 18, 2023. Accessed May 25, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-moderately-severely-active-crohns-disease.