Top Recent FDA Approvals

Recent FDA approvals include treatments for acute low back pain, postoperative nausea and vomiting, multiple myeloma, hemophilia B, and fibromyalgia pain.

The US Food and Drug Administration (FDA) approved several drugs relevant to conditions often treated by nurse practitioners and PAs. Approvals include treatments for hemophilia B, acute low back pain, postoperative nausea and vomiting, multiple myeloma, and fibromyalgia pain.

Gene Therapy for Hemophilia B

The first gene therapy for adults with hemophilia B (congential Factor IX deficiency) has been approved by the FDA.  Hemgenix® is an adeno-associated virus vector-based gene therapy for adults who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. The single dose treatment generates factor IX proteins that are 5 to 8 times more active than normal.

The approval was based on data from the HOPE-B trial that included 54 male participants with severe or moderately severe hemophilia B. After the lead-in period, patients received a single intravenous dose of etranacogene dezaparvovec and entered a follow-up period of 5 years. The primary endpoint was a noninferiority test of annualized bleeding rate (ABR) during months 7 to 18 after etranacogene dezaparvovec treatment compared with the 6-month lead-in period.

Results showed that the estimated mean ABR was 1.9 bleeds/year (95% CI, 1.0-3.4) 7 to 18 months postinfusion compared with an estimated mean ABR of 4.1 bleeds/year (95% CI, 3.2-5.4) during the lead-in period. The ABR ratio was reported to be 0.46 (95% CI, 0.26-0.81), demonstrating noninferiority of ABR during months 7 to 18 vs the lead-in period.

Additionally, 94% (n=51/54) of participants treated with etranacogene dezaparvovec discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy.

New Strength of ZTildo® for Acute Low Back Pain

The FDA has fast tracked SP-103 (lidocaine topical system) 5.4%, a next generation triple strength formulation of ZTlido® for the treatment of acute low back pain. The investigational agent delivers 108 mg lidocaine using the same nonaqueous, adhesive drug delivery system as ZTlido 1.8%, which is approved for relief of pain associated with postherpetic neuralgia.

The safety and efficacy of SP-103 is currently being evaluated in patients diagnosed with moderate to severe acute low back pain in a randomized, double-blind, placebo-controlled phase 2 trial. The trial will be completed within the first quarter of 2023.

Diacomit® for Younger Patients With Dravet Syndrome

The FDA has expanded the approval of Diacomit® (stiripentol)to include patients 6 months and older weighing 7 kg or more diagnosed with seizures associated with Dravet syndrome who are taking clobazam.

Effectiveness of stiripentol in this younger age group was based on extrapolation of data from patients 3 years to younger than 18 years of age in 2 multicenter, placebo-controlled, double-blind, randomized clinical studies.

The most commonly adverse reactions reported are somnolence, decreased appetite, agitation, ataxia, weight decreased, hypotonia, nausea, tremor, dysarthria, and insomnia.

Stiripentolis supplied as 250 mg and 500 mg strength capsules in 60-count bottles and as fruit-flavored powder packets for oral suspension in 60-count cartons.

Aponviefor Postoperative Vomiting and Nausea

The FDA approved Aponvie™ (aprepitant), an intravenous injectable emulsion formulation, for the prevention of postoperative nausea and vomiting (PONV). The drug was designed to directly deliver aprepitant, a substance P/neurokinin-1 (NK1) receptor antagonist, as a 30-second IV injection.

Approval of Aponvie™ is based on data obtained from 2 multicenter, randomized, double-blind clinical studies comparing oral aprepitant to current standard of care, IV ondansetron, for the prevention of PONV in patients during the 48 hours after open abdominal surgery. Approximately 50% fewer patients treated with the Aponvie experienced postoperative vomiting in the first 24 and 48 hours compared with those who received ondansetron.

Aponvie was well tolerated with a comparable safety profile to oral aprepitant. Aponvie is supplied in a single-dose vial containing 32 mg of aprepitant.

Tecvaylifor Relapsed or Refractory Multiple Myeloma

The FDA has granted accelerated approval for Tecvayli (teclistamab-cqyv) for the treatment of relapsed or refractory multiple myeloma in adults who previously received 4 or more prior lines of therapy, including a proteasome inhibitor, immunomodulatory drug and anti-CD38 monoclonal antibody.

The approval was based on data obtained from the single-arm, open-label, multicenter MajesTEC-1 study that evaluated the safety and efficacy of teclistamab-cqyv in 110 adults diagnosed with relapsed or refractory multiple myeloma who had received at least 3 prior therapies. Patients received teclistamab-cqyv via subcutaneous injection at step-up doses of 0.06 mg/kg and 0.3 mg/kg followed by 1.5 mg/kg once weekly.

Findings indicated a response rate of 61.8% (95% CI, 52.1-70.9); 28.2% of patients achieved complete response or better, 29.1% achieved very good partial response, and 4.5% achieved partial response.

Tecvaylicarries a boxed warning for life threatening or fatal cytokine release syndrome (CRS) and neurologic toxicity, including immune effector cell-associated neurotoxicity. 

The most common adverse reactions included were pyrexia, CRS, musculoskeletal pain, injection site reaction, fatigue, upper respiratory tract infection, nausea, headache, pneumonia, diarrhea, and laboratory abnormalities.

Tecvayli is supplied as 30 mg/3 mL and 153 mg/1.7 mL of teclistamab-cqyv in single-dose vials.

Single-Vial Presentation of Meningococcal Vaccine Menveo

A ready-to-use single dose vial of Menveo (meningococcal [Groups A, C, Y, W-135] oligosaccharide diphtheria CRM197 conjugate vaccine) was approved by the FDA for immunization against invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, Y and W-135 in patients 10 to 55 years of age. 

Approval was based on data obtained from an observer-blind randomized, multicenter controlled phase 2 trial that compared the safety and immunogenicity of Menveo 1-vial presentation to the Menveo 2-vial presentation in individuals 10 to 40 years of age. 

Results indicated noninferiority of MenA serogroup hSBA GMTs at 28 days postvaccination for the Menveo 1-vial group compared with the Menveo 2-vial presentation group. 

Menveo 1-vial presentation is expected to be available in 2023. Menveo is currently available in the 2-vial presentation, which requires reconstitution before use. 

FibroLux Laser Therapy for Treatment of Fibromyalgia Pain

FibroLux is a laser-based therapy that was approved by the FDA for adjunctive use in the temporary relief of pain as a result of fibromyalgia. The laser therapy delivers optimal doses of light energy using a combination of curated wavelengths.

Approval was based on data obtained from a placebo-controlled, randomized, double-blind, group clinical study that included 90 patients diagnosed with fibromyalgia. Patients were randomly assigned to receive either FibroLux or placebo 3 times a week for a total of 9 treatment sessions over a 3-week period. 

Results indicated that the majority of participants treated with FibroLux had a clinically meaningful and statistically significant decrease in tender point count (TPC of 20% or greater) compared with those who received placebo (primary endpoint; 86.67% vs 48.89%, respectively; treatment difference, 37.78%; P <.0005).

This article originally appeared on Clinical Advisor


1. Hemgenix®. Package insert. CSL Behring; 2022. Accessed November 22, 2022.

2. ZTlido®. Prescribing information. Scilex Holding Company; 2022. Accessed November 15, 2022.

3. Diacomit®. Prescribing information. Biocodex, Inc.; 2022. Accessed November 15, 2022.

4. Aponvie. Prescribing information. Heron Therapeutics, Inc.; 2022. Accessed November 15, 2022.

5. Tecvayli. Prescribing information. Janssen Biotech, Inc.; 2022. Accessed November 15, 2022.

6. Menveo. Prescribing information. GlaxoSmithKline plc; 2022. Accessed November 15, 2022.

7. Multi Radiance Medical therapeutic laser receives FDA clearance for the treatment of pain associated with fibromyalgia. News release. Multi Radiance Medical. Accessed November 15, 2022.