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Aug 20, 2023

Recent 2023 FDA Approvals

The US Food and Drug Administration (FDA) approved drugs in April and May 2023

The US Food and Drug Administration (FDA) approved drugs in April and May 2023 relevant to conditions often treated by nurse practitioners and PAs. Approvals include treatments for chronic migraine, type 1 diabetes, blood cancers, tardive dyskinesia and chorea associated with Huntington disease, vasomotor symptoms of menopause, pain and fever, and Fabry disease.

The FDA expanded the approval of the oral calcitonin gene-related peptide (CGRP) receptor antagonist Qulipta® (atogepant) to include the preventive treatment of chronic migraine in adults. Qulipta was previously only approved to prevent episodic migraine.

Approval was based on data from the randomized, double-blind, placebo-controlled phase 3 PROGRESS study. Participants had at least a 1-year history of chronic migraine with a mean of 19 migraine days per month at baseline. Participants were randomly assigned atogepant 60 mg once daily (n=262) or placebo (n=259) for 12 weeks.

Results indicated that patients assigned to atogepant experienced a significant reduction from baseline in mean monthly migraine days of 6.9 days compared with 5.1 days in those assigned placebo (P <.001). The proportion of patients achieving at least a 50% reduction from baseline in mean monthly migraine days was greater in the atogepant group (41% vs 26%; P <.001)

The most common adverse reactions reported were constipation, nausea, and fatigue/sleepiness.

Qulipta is available as 10-mg, 30-mg, and 60-mg tablets in 30-count bottles; only the 60-mg dose is indicated for the preventive treatment of chronic migraine.

The FDA has cleared the Beta Bionics iLet ACE Pump and the iLet Dosing Decision Software for individuals 6 years of age and older with type 1 diabetes. When combined with a integrated continuous glucose monitor (iCGM), the iLet ACE pump and software form an automated insulin dosing system called the iLet Bionic Pancreas.

The iLet Bionic Pancreas uses an adaptive closed-loop algorithm requiring only a user's body weight for start-up and no additional insulin dosing parameters. The device also includes a new meal announcement feature that allows users to estimate the amount of carbs in their meal as small, medium, or large. The adaptive algorithm will learn over time to respond to the users’ individual insulin needs.

The FDA approved the Medtronic MiniMedTM 780G system with the GuardianTM 4 sensor for patients aged 7 years and older diagnosed with type 1 diabetes. The device is designed for continuous glucose monitoring in addition to delivering basal insulin at selectable rates and issuing correction insulin boluses without user input.

With the Company's SmartGuardTM technology, the MiniMed 780G can be programmed to adjust insulin delivery.

Approval was based on data from a single-arm study that included 157 participants (aged 14 to 75 years) with type 1 diabetes. Treatment led to a reduction in HbA1C and increased overall time in range compared with a run-in period that included a sensor-augmented pump with or without predictive low glucose management or automated basal insulin delivery use.

MiniMed 780G is expected to be available in the summer with preorders beginning in May 2023. Patients using the MiniMed 770G will be eligible to upgrade their device to the MiniMed 780G remotely at no cost.

Omisirge® (omidubicel-onlv) was approved by the FDA for use in adults and pediatric patients aged 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.

The treatment is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood. Treatment is administered as a single intravenous dose; each dose is patient-specific and contains healthy stem cells from an allogeneic prescreened donor.

The approval was based on data from a multicenter, randomized, phase 3 trial that included patients 12 to 65 years of age with hematologic malignancies who are eligible for allogeneic transplantation.

Results indicated that 87% of patients who received omidubicel-onlv achieved neutrophil recovery compared with 83% of those who received standard umbilical cord blood transplantation (UCBT). Grade 2/3 bacterial or grade 3 fungal infections through day 100 following transplantation occurred in 39% of patients in the omidubicel-onlv arm and 60% of patients in the UCBT arm.

The most common adverse reactions reported were infections, graft vs host disease, and infusion reactions. Omisirge labeling includes a Boxed Warning regarding the risk of infusion reactions, graft vs host disease, engraftment syndrome, and graft failure. Because of the potential for severe adverse reactions, health care professionals should assess the risks and benefits of using the treatment.

Austedo® XR (deutetrabenazine) extended-release tablets were approved by the FDA to treat tardive dyskinesia and chorea associated with Huntington disease in adult patients.

Austedo XR is a vesicular monoamine transporter 2 inhibitor. The once-daily formulation was found to be therapeutically equivalent to the twice-daily formulation. Unlike Austedo (twice daily) tablets, which should be administered with food, the new once-daily formulation can be administered with or without food.

Austedo XR is supplied in 6-mg, 12-mg, and 24-mg strength extended-release tablets in 30-count bottles. The dose is determined individually for each patient based on the reduction of chorea or tardive dyskinesia and tolerability.

The FDA approved VeozahTM (fezolinetant)for the treatmentof moderate to severe vasomotor symptoms of menopause. Veozah is an oral neurokinin 3 (NK3) receptor antagonist and works by blocking neurokinin B binding on the kisspeptin/neurokinin/dynorphin neuron to regulate neuronal activity in the thermoregulatory center.

Approval was based on the BRIGHT SKY program that included safety and efficacy data from the phase 3 SKYLIGHT 1 and 2 studies along with the SKYLIGHT 4 safety study. The SKYLIGHT 1 and 2 studies evaluated the efficacy and safety of fezolinetant (30 mg and 45 mg) orally once daily in 1022 women (40 to 65 years old) with moderate to severe vasomotor symptoms.

Results showed a statistically significant and clinically meaningful reduction from baseline in the frequency of moderate to severe vasomotor symptoms with 45 mg at weeks 4 and 12. The 45-mg dose was also associated with a statistically significant reduction from baseline in the severity of moderate to severe vasomotor symptoms (over 24 hours) compared with placebo at weeks 4 and 12.

The most common adverse reactions reported with fezolinetant were abdominal pain, diarrhea, insomnia, back pain, hot flush, and hepatic transaminase elevation.

Veozah is supplied as 45-mg tablets in 30- and 90-count bottles. The treatment is taken once a day at the same time each day.

Caldolor® (ibuprofen; Cumberland Pharmaceuticals) Injection was approved by the FDA for the treatment of pain and fever in pediatric patients 6 months of age and older. This is the first nonsteroidal anti-inflammatory drug (NSAID) approved for use in pediatric patients.

The approval was based on data from clinical trials that evaluated the safety and efficacy of this agent in hospitalized febrile children and children undergoing tonsillectomy surgery. A statistically significant greater reduction in temperature was seen in pediatric patients given Caldolor vs acetaminophen; 74% of patients treated with Caldolor became afebrile by the end of the first dosing interval.

The most common side effects reported in pediatric patients were infusion site pain, vomiting, nausea, anemia, and headache. Caldolor must be diluted before administration.

The drug is also approved for adults in the management of mild to moderate pain and moderate to severe pain as an adjunct to opioid analgesics, as well as for the reduction of fever.

The FDA approved Elfabrio® (pegunigalsidase alfa-iwxj) to treat adults diagnosed with Fabry disease. Elfabrio is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme and is internalized and transported into lysosomes where it is thought to exert enzymatic activity and reduced accumulated globotriaosylceramide (Gb3) in blood vessel walls throughout the body.

Approval was based on data from 2 clinical trials: an open-label dose-ranging study in patients who were enzyme replacement therapy (ERT)-naïve or who had not received ERT for more than 26 weeks and had a negative test for anti-pegunigalsidase alfa-iwxj IgG antibodies; and a phase 3, double-blind, and active-controlled trial in ERT-experienced adults diagnosed with Fabry disease.

In the phase 3 trial, patients were randomly assigned to receive at least 1 dose of pegunigalsidase alfa (n=52) or agalsidase beta (n=25).

Findings showed that pegunigalsidase alfa was noninferior to agalsidase beta in controlling eGFR decline. The estimated mean eGFR slope was -2.4 and -2.3 mL/min/1.73 m2/year on pegunigalsidase alfa and agalsidase beta respectively. The estimated treatment difference was -0.1 mL/min/1.73 m2/year (95% CI, -2.3, 2.1).

The most common adverse reactions reported were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.

Elfabrio is supplied as a preservative-free solution in a single-dose vial. Each vial contains 20 mg/10 mL of pegunigalsidase alfa-iwxj. Treatment is administered by intravenous infusion every 2 weeks.

1. Qulipta. Prescribing information. AbbVie; 2023. Accessed May 22, 2023. https://www.rxabbvie.com/pdf/QULIPTA_pi.pdf

2. FDA clears new insulin pump and algorithm-based software to support enhanced automatic insulin delivery. News release. US Food and Drug Administration. Accessed May 19, 2023. https://www.prnewswire.com/news-releases/fda-clears-new-insulin-pump-and-algorithm-based-software-to-support-enhanced-automatic-insulin-delivery-301829765.html

3. FDA approves Medtronic MiniMed™ 780G System – world's first insulin pump with meal detection technology featuring 5-minute auto corrections. News release. Medtronic. May 22, 2023. https://www.prnewswire.com/news-releases/fda-approves-medtronic-minimed-780g-system—worlds-first-insulin-pump-with-meal-detection-technology-featuring-5-minute-auto-corrections-301804565.html.

4. Omisirge. Prescribing information. Gamida Cell; 2023. Acceessed May 22, 2023. http://www.gamida-cell.com/wp-content/uploads/Omisirge-final-PI.pdf

5. Austedo XR. Prescribing information. Teva Pharmaceuticals; 2023. Accessed May 22, 2023. https://www.austedo.com/globalassets/austedo/prescribing-information.pdf

6. Veozah. Prescribing information. Astellas Pharma Inc.; 2023. Accessed May 22, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216578s000lbl.pdf

7. Caldolor. Prescribing information. Cumberland Pharmaceuticals; 2023. Accessed May 22, 2023. https://cumberlandpharmaceuticalsinc.gcs-web.com/node/14576/pdf

8. Elfabrio. Prescribing information. Chiesi; 2023. Accessed May 22, 2023. https://resources.chiesiusa.com/Elfabrio/ELFABRIO_PI.pdf