Here is information on the latest US FDA approvals, the week of July 24 – July 28, 2023.
RiVive OTC Approval for Opioid Overdose
The FDA has approved RiVive, a 3 mg naloxone hydrochloride nasal spray, for over-the-counter (OTC) use in the emergency treatment of known or suspected opioid overdose. The FDA granted the nonprescription approval of RiVive to the manufacturer, Harm Reduction Therapeutics. RiVive rapidly reverses or blocks the effects of opioids, restoring normal respiration, especially when given within minutes of the first signs of an overdose. This week’s approval marks the second nonprescription naloxone product, the other being Narcan, manufactured by Emergent BioSolutions, approved for OTC use on March 29, of this year. Overdose remains a significant public health concern in the United States, particularly with respect to synthetic opioids such as illicit fentanyl, with more than 105,000 reported fatal overdoses occurring in the 12-month period ending in February 2023. Naloxone rapidly reverses opioid overdose effects. The approval of RiVive for nonprescription use was supported by data demonstrating its effectiveness and safety, as well as consumers’ ability to use it correctly without the supervision of a healthcare professional, although RiVive may cause severe opioid withdrawal symptoms in individuals dependent on opioids.
Balfaxar for Urgent Reversal of Warfarin (VKA) Therapy
The FDA has approved Balfaxar® (prothrombin complex concentrate, human-lans), used for urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist therapy, such as warfarin, in adult patients requiring urgent surgery or invasive procedures. Balfaxar®, manufactured by Octapharma USA, replenishes deficient clotting factors caused by warfarin therapy and is a non-activated 4-factor prothrombin complex concentrate (4F-PCC) containing vitamin K-dependent factors. The approval was supported by the LEX-209 clinical trial, which demonstrated this agent’s hemostatic efficacy and non-inferiority compared to a comparator, Kcentra®. Over 2.4 million patients are prescribed warfarin each year in the US, which increases bleeding risk during urgent medical procedures and may require hemostatic treatment such as Balfaxar. The prescribing information for Balfaxar includes a boxed warning for risk of thromboembolic events, especially in patients with the history of a thromboembolic events.
https://balfaxar.com/wp-content/uploads/2023/07/Balfaxar_Prescribing_Information_072623.pdf
Xdemvy for Demodex Blepharitis
The FDA has approved XdemvyTM (lotilaner ophthalmic solution 0.25%), developed by Tarsus Pharmaceuticals, as the first treatment for Demodex blepharitis. Demodex blepharitis is a common eyelid disease affecting about 25 million patients in the United States. The condition is often misdiagnosed or underdiagnosed and is characterized by redness, inflammation, missing or misdirected eyelashes, itching along the eyelid base and the presence of waxy debris waste products found at the base of the eyelashes, called collarettes. Xdemvy directly targets inflammation caused by Demodex mites, which are microscopic parasites living in hair follicles and oil glands. Almost everyone has these mites, but they usually don’t cause problems. However, they can proliferate in people who are immunocompromised or have other skin conditions. Administered as a single drop in each eye twice daily for 6 weeks, Xdemvy eradicates the mites, which thankfully, are too small to be seen with the naked eye. Clinical trials demonstrated the efficacy of Xdemvy, with patients experiencing improvement within two weeks. The treatment was generally safe and well-tolerated, with common ocular adverse reactions being eye stinging and burning in about 10% of patients. Xdemvy will be available by the end of August 2023.
https://ir.tarsusrx.com/news-releases/news-release-details/fda-approves-xdemvytm-lotilaner-ophthalmic-solution-025
https://tarsusrx.com/wp-content/uploads/XDEMVY-Prescribing-Information-24JUL23.pdf
Upcoming PDUFA dates
Just in case definition, the FDA PDUFA (Prescription Drug User Fee Act) date is the target deadline for the FDA to complete its review of a New Drug Application (NDA) or Biologics License Application (BLA) submitted by a pharmaceutical company.
- Remestemcel-L for aGVHD Remestemcel-L is being developed for inflammatory diseases in children and adults including the treatment of acute graft versus host disease (aGVHD), a potentially life-threatening complication of an allogeneic bone marrow transplant (BMT) and has a PDUFA date of Wednesday of this week, August 2. According to press release from the manufacturer, Mesoblast, “Currently, there are no products approved in the United States for treatment of steroid-refractory aGVHD in children under 12, and off-label options have demonstrated mixed efficacy with high toxicity.” Remestemcel-L is derived from culture-expanded mesenchymal stromal cells from donor bone marrow and is thought to counteract cytokine storms that can occur allogeneic bone marrow transplant. The FDA issued a complete response letter (CRL) to Mesoblast Limited, in October 2020, requesting additional data from at least 1 randomized, controlled study in adult and/or pediatric patients with steroid-refractory aGVHD.
https://www.mesoblast.com/product-candidates/oncology-hematology/acute-graft-versus-host-disease
https://investorsmedia.mesoblast.com/static-files/1e259fcb-77ba-470c-91af-1c71e5fa32e0
- Zuranolone for Depression
The PDUFA date for zuranolone is set for this Saturday, August 5, 2023 after the NDA was accepted for priority review in February of this year. Zuranolone (SAGE-217), manufactured by Biogen Inc and Sage Therapeutics is a potential treatment for major depressive disorder (MDD) and postpartum depression (PPD). Zuranolone has been assessed as a once-daily, 14-day oral regimen in adults with MDD or PDD. The NDA includes data from the NEST and LANDSCAPE clinical development programs and a phase 2 study from Japan. Zuranolone is a neuroactive steroid and has a novel mechanism of action as a positive allosteric modulator of GABA-A receptors. In people with depression, zuranolone is thought to work by rapidly rebalancing dysregulated neuronal networks to help reset brain function.
Please check back every Monday morning for last week’s approvals so that you can stay up to date.
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