Drug Approvals Monthly Update: May 2024

May 30, 2024

This monthly update of United States (U.S.) Food and Drug Administration (FDA) approvals provides a review of newly approved specialty drugs, new indications and recent first-time generic launches.


New drugs

04/22/2024 Anktiva® (nogapendekin alfa inbakicept-pmln)

The FDA approved Anktiva for use in combination with Bacillus Calmette-Guérin (BCG) for the treatment of adults with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Anktiva is a first-in-class interleukin-15 superagonist that was granted Breakthrough Therapy and Fast Track designations by the FDA. Anktiva is approved as a solution that is instilled into the bladder (intravesically) with BCG for up to 2 hours. Induction treatment is given once a week for 6 weeks (may be repeated if needed) and maintenance therapy is given once a week for 3 weeks at months 4, 7, 10, 13, and 19, with additional doses allowed in months 25, 31, and 37. In the single-arm QUILT-3.032 study, Anktiva led to a complete response rate (CRR) of 62%. Among patients who achieved a complete response (CR), 58% had duration of response (DOR) ≥ 12 month, and 40% had DOR ≥ 24 months. Anktiva may compete with Adstiladrin® (nadofaragene firadenovec) and Keytruda® (pembrolizumab) in the BCG-unresponsive NMIBC space.¹ ImmunityBio launched Anktiva in mid-May 2024, at $35,800 per dose.

04/23/2024 Ojemda (tovorafenib)

Day One received an Accelerated Approval for their oral, type II RAF kinase inhibitor, Ojemda, for the treatment of patients 6 months of age and older with relapsed or refractory (R/R) pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. Continued approval may depend upon results from a confirmatory trial(s). Ojemda is taken by mouth once weekly. It was granted Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations and a Priority Review by the FDA. In the open-label, phase 2 FIREFLY-1 trial, Ojemda led to an overall response rate (ORR) of 51%, with a partial response rate of 28% and minor response rate of 11%; no CRs were reported.² In addition, the ORR was 52% in patients with BRAF fusion or rearrangement, and 50% among patients with BRAF V600E mutation. The median time to response was 5.3 months and median DOR was 13.8 months. Ojemda will serve as an alternative to dabrafenib (Tafinlar®) plus trametinib (Mekinist®) or monotherapy with vemurafenib (Zelboraf®; off-label) for the treatment of BRAF V600E-mutated tumors in pediatric patients with R/R pLGG. Ojemda is available in the U.S. through the specialty pharmacies Biologics by McKesson and Onco360® at a wholesale acquisition cost (WAC) of $33,916 for a 28-day supply.

04/26/2024 Beqvez (fidanacogene elaparvovec-dzkt)

The FDA approved Beqvez for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. Beqvez received Breakthrough Therapy, Orphan Drug and Regenerative Medicine Advanced Therapy designations. Administered as a one-time intravenous (IV) infusion, Beqvez introduces a functional copy of the FIX gene and enables patients with hemophilia B to produce adequate levels of FIX. In the phase 3 BENEGENE-2 trial, Beqvez was superior to prophylactic exogenous FIX. It reduced the annualized bleeding rate (ABR) by 71% during weeks 12 weeks to 15 months in adult men with hemophilia B. Beqvez is the second gene therapy approved for hemophilia B, following Hemgenix® (etranacogene dezaparvovec-drlb) in November 2022. The FDA also approved Labcorp’s nAbCyte Anti-AAVRh74var HB-FE Assay as a companion diagnostic to determine eligibility for Beqvez treatment. Pfizer has launched Beqvez at a price of $3.5 million for the one-time dose.

04/26/2024 Xolremdi (mavorixafor)

Following a Priority Review, X4 Pharmaceuticals’ chemokine receptor (CXCR4) antagonist, Xolremdi, was FDA-approved for the treatment of warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome to increase the number of circulating mature neutrophils and lymphocytes in patients aged 12 and older. Xolremdi is the first medication approved for WHIM syndrome, a rare, inherited disorder characterized by neutropenia, lymphopenia, and serious and/or frequent infections. Approved as oral capsules, Xolremdi is taken once daily on an empty stomach at least 30 minutes before consuming the first food of the day. In the phase 3, double-blind, 52-week 4WHIM trial, Xolremdi led to an increased time above threshold for absolute neutrophil count (≥ 500 cells/microliter) over 24 hours and time above threshold for absolute lymphocyte count (≥ 1000 cells/microliter) over 24 hours compared to placebo (p<0.0001 for both).³ In addition, Xolremdi resulted in an approximate 40% reduction in total infection score, weighted by infection severity, compare to placebo. The FDA granted Xolremdi Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for WHIM syndrome. Xolremdi is available in the U.S. through the specialty pharmacy PantherRx® Rare. The annual WAC ranges from $372,300 to $496,400, depending on patient weight.

05/16/2024 Imdelltra (tarlatamab-dlle)

The FDA granted an Accelerated Approval for Imdelltra for the treatment of adults with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. Continued approval may depend on results from a confirmatory trial(s). Imdelltra a first-in-class bispecific delta-like ligand 3 (DLL3)-directed CD3 T-cell engager (BiTE), providing a new approach to combat SCLC. It was granted Breakthrough Therapy and Orphan Drug designations by the FDA. Imdelltra is administered via IV infusion. Following the initial three step-up doses, it is administered every 2 weeks until disease progression or unacceptable toxicity. The agent carries a boxed warning for cytokine release syndrome and neurologic toxicity. Therefore, it requires patient monitoring for approximately 24 hours during and after the first 2 doses and patients should remain within 1 hour of the health care setting for a total of 48 hours. Observation periods decrease for subsequent doses. In the phase 2 DeLLphi-301 trial, the approved dosage demonstrated an ORR of 40%, with a CRR of 2%.4 The median DOR was 9.7 months (range, 2.7 to 20.7) and median overall survival (OS) was 14.3 months, with final and complete survival data yet to mature. Amgen has set the U.S. price for Imdelltra at $31,500 for the first 28-day cycle (step-up dosing), and $30,000 for subsequent cycles. The company noted that patients were treated for a median of 5.5 cycles in clinical trials; therefore, the average cost per patient is estimated at $166,500.

New biosimilars

04/25/2024 Hercessi (trastuzumab-strf)

Accord announced the FDA approved Hercessi as a biosimilar to Genentech’s trastuzumab (Herceptin®), a human epidermal growth factor receptor 2 (HER2)/neu receptor antagonist. Hercessi is indicated for the treatment of HER2-overexpressing breast cancer and HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma. Hercessi is approved as a lyophilized powder for reconstitution prior to IV administration. Recommended dosage regimens for HER2-overexpressing breast cancer and metastatic HER2-overexpressing gastric cancer align with Herceptin. Hercessi is anticipated to be available in the U.S. in 1H 2024, with pricing to follow. A 420 mg-strength presentation of Hercessi is also in development with an FDA decision anticipated in 4Q 2024.

04/30/2024 Cyltezo® (adalimumab-adbm) high-concentration

The FDA approved a new high-concentration formulation of Boehringer Ingelheim’s Cyltezo (100 mg/mL) in a 40 mg/0.4 mL prefilled syringe and 40 mg/0.4 mL prefilled autoinjector. Cyltezo was already approved as a low-concentration formulation (50 mg/mL) in a prefilled pen and prefilled syringe (both 40 mg/0.8 mL). The FDA also granted Cyltezo interchangeable status to Abbvie’s Humira® (adalimumab).

5/20/2024 Opuviz (aflibercept-yszy) and Yesafili (aflibercept-jbvf)

Samsung Bioepis/Biogen and Biocon received FDA approval for Opuviz and Yesafili, respectively, as interchangeable biosimilars to Regeneron’s vascular endothelial growth factor (VEGF) inhibitor Eylea® (aflibercept). Opuviz and Yesafili are indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy. Unlike Eylea, neither product is indicated for retinopathy of prematurity. Opuviz and Yesafili are approved as 2 mg/0.05 mL solutions in single-dose vials. Launch timeframe for both biosimilars are to be determined due to ongoing litigation. Pricing to follow.

New indications

04/18/2024 Alecensa® (alectinib)

The FDA approved a new indication for Genentech’s kinase inhibitor, Alecensa, for the adjuvant treatment in adults following tumor resection of anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) (tumors ≥ 4 cm or node positive), as detected by an FDA-approved test. Alecensa is administered orally twice daily, for a total of 2 years or until disease recurrence or unacceptable toxicity.

04/18/2024 Entyvio® (vedolizumab)

The FDA approved an expanded indication for the subcutaneous (SC) formulation of Entyvio, by Takeda, for the treatment of adults with moderately to severely active Crohn’s disease (CD). Treatment for CD is initiated as 300 mg administered IV at weeks 0 and 2. At week 6 and beyond the 300 mg IV dose is administration every 8 weeks. Alternatively, at week 6 or beyond, the patient may switch to using the SC formulation at a dose of 108 mg once every 2 weeks. Entyvio is also indicated for the treatment of adults with ulcerative colitis using the same IV and SC dosage regimens. A health care professional administers IV doses. SC doses may be administered by the patient or caregiver, using the prefilled syringe or prefilled pen, with proper training.

4/30/2024 Otezla® (apremilast)

Amgen received an expanded indication for Otezla to include the treatment of pediatric patients 6 to 17 years of age weighing ≥ 20 kg with moderate to severe plaque psoriasis (PSO) who are candidates for phototherapy or systemic therapy. Otezla was previously only approved for treatment of PSO in adults. The recommended dosage for pediatric patients is weight-based.

05/15/2024 Benlysta (belimumab)

The FDA approved an expanded indication for GlaxoSmithKline’s B-lymphocyte stimulator (BLyS)-specific inhibitor, Benlysta, for SC administration via the autoinjector to include treatment of pediatric patients 5 years and older with active systemic lupus erythematosus (SLE) who are receiving standard therapy. Previously, only Benlysta via IV administration was approved for use in pediatric patients with SLE.

05/15/2024 Breyanzi® (lisocabtagene maraleucel)

The FDA granted an Accelerated Approval for Breyanzi, by Bristol-Myers Squibb, for the treatment of adults with R/R follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Breyanzi is the third CD19-directed chimeric antigen receptor T cell (CAR-T) therapy approved for this indication. Approval was based on the phase 2, open-label, single-arm TRANSCEND-FL trial, which demonstrated an ORR of 95.7% with 1 dose of Breyanzi. The median DOR was not reached after a median follow-up of 16.8 months.5


New drugs

04/19/2024 Rezenopy (naloxone hydrochloride) nasal spray

A high-dose formulation of naloxone nasal spray, Rezenopy, by Summit Biosciences, was approved under a 505(b)(2) new drug application (NDA) for the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and/or central nervous system depression in adults and pediatric patients. Rezenopy is approved as a 10 mg/0.11 mL dose in a single-spray device; with 2 devices provided per carton. Naloxone nasal spray is also available as 3 mg (Rivive), 4 mg (Narcan®), and 8 mg (Kloxxado®) doses. Unlike the lower-dose formulations, the Rezenopy label advises against administering more than two sprays per day.6 Rezenopy and Kloxxado require a prescription, while Rivive and Narcan nasal spray are available over-the-counter. Summit Biosciences has not announced the launch timeframe or pricing for Rezenopy.

04/24/2024 Pivya (pivmecillinam)

Utility Therapeutics received FDA approval of Pivya for the treatment of women 18 years of age and older with uncomplicated urinary tract infections (uUTI) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus. Pivya was granted a Qualified Infectious Disease Product (QIDP) designation and a Priority Review by the FDA. It was approved as oral tablets to be taken three times a day for 3 to 7 days as clinically indicated. Pivya demonstrated efficacy for treating uUTI across three separate clinical trials based on the composite response rate, consisting of clinical cure (resolution of baseline uUTI symptoms) and microbiological response (reduction of urine bacteria as seen on culture). In the first trial, composite response rate was 62% with Pivya compared to 10% for placebo.7 The second trial reported a composite response rate of 72% with Pivya compared to 76% with the antibiotic cephalexin. In the third trial, composite response was achieved in 66% of patient who received Pivya compared to 22% who received ibuprofen. There continues to be a need for new antibiotics to treat infection due to the development of drug resistant bacterial strains. Alternative treatment options for uUTI include sulfamethoxazole/trimethoprim, nitrofurantoin, and fluoroquinolones. Launch of Pivya is anticipated in 2025; pricing will follow.

4/29/2024 Libervant (diazepam) buccal film

The FDA approved Libervant under their 505(b)(2) NDA pathway for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (e.g., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy 2 to 5 years of age. It is approved as a buccal film. No more than 2 doses should be used to treat a single episode. In addition, Libervant should not be used to treat more than one episode every 5 days or more than 5 episodes per month. As with other benzodiazepine agents, Libervant is a scheduled IV controlled substance. Aquestive Therapeutics anticipates launching Libervant in the U.S. in January 2027.

4/30/2024 Ingrezza® Sprinkle (valbenazine)

The FDA approved a new sprinkle formulation for Ingrezza, by Neurocrine Biosciences. Ingrezza is indicated for the treatment of adults with tardive dyskinesia and chorea associated with Huntington’s disease. Ingrezza Sprinkle is approved as 40 mg, 60 mg, and 80 mg capsules that may be opened and sprinkled over soft food or swallowed whole with water. Ingrezza Sprinkle is appropriate for patients who experience dysphagia or have difficulty swallowing.

05/01/2024 Myhibbin (mycophenolate mofetil) oral suspension

The FDA approved Myhibbin under the 505(b)(2) approval pathway. Myhibbih is a ready-to-use mycophenolate mofetil oral suspension, indicated for the prophylaxis of organ rejection in adult and pediatric recipients 3 months of age and older with allogeneic kidney, heart, or liver transplants, in combination with other immunosuppressants. Myhibbin is anticipated to be available in 2Q 2024. Azurity has not provided pricing information.

First generic drug launches

Estrogel® (estradiol gel): Soliris launched its generic version of Ascend’s Estrogel transdermal metered gel, an estrogen agent indicated for the treatment of moderate to severe vasomotor symptoms due to menopause. It is not evident if additional companies are seeking approval for Estrogel generics. Estrogel generated $17 million in annual U.S. sales in 2023.

Halaven® (eribulin mesylate): After a settlement agreement, Gland Pharmaceuticals launched its generic version of Halaven injection, a microtubule inhibitor indicated for treatment of select patients with breast cancer or liposarcoma. There is potential for three additional companies to launch generic eribulin mesylate in 2024-2025, if approved by the FDA. Halaven generated $108 million in annual U.S. sales in 2023.

Myrbetriq® (mirabegron): Lupin and Zydus launched generic versions of Astellas’ Myrbetriq tablets in 50 mg (Zydus) and/or 25 mg (Lupin and Zydus) strengths. Mirabegron is a beta-3 adrenergic agonist indicated for treatment of overactive bladder and neurogenic detrusor overactivity in select patients. There is potential for several other generics to Myrbetriq tablets to enter the U.S. market. Myrbetriq tablets generated $2.861 billion in annual U.S. sales in 2023.

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