Drug Approvals Monthly Update: June 2024

June 28, 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.

Specialty

New drugs

06/06/2024 Rytelo™ (imetelstat)

The FDA approved the first-in-class oligonucleotide telomerase inhibitor, Rytelo, for the treatment of adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring ≥ 4 red blood cell (RBC) units over 8 weeks who have not responded to or have lost response to, or are ineligible for, erythropoiesis-stimulating agents (ESA). Rytelo was granted Breakthrough Therapy and Orphan Drug designations by the FDA. This drug is administered as an intravenous (IV) infusion every 4 weeks. Treatment should be stopped if RBC transfusion burden does not decrease after 24 weeks (6 doses) or if the patient experiences unacceptable toxicity. In the double-blind, phase 3 IMerge trial (n=178), treatment with Rytelo led to a 40% rate of ≥ 8-week RBC transfusion independence compared to 15% with placebo.¹ Rytelo may compete with luspatercept-aamt (Reblozyl®) in the MDS treatment space. According to its manufacturer, Geron, the Rytelo 188 mg vial is expected to be available by the end of June 2024 at an average wholesale price (AWP) of $9,884 per vial, and the 47 mg vial is anticipated to launch later in the summer at $2,471 per vial.

06/10/2024 Iqirvo® (elafibranor)

Ipsen received Accelerated Approval for their oral, first-in-class peroxisome proliferator-activated receptor (PPAR) agonist, Iqirvo, for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. Continued approval may depend upon results from a confirmatory trial(s). Iqirvo was approved under Priority Review and was granted Breakthrough Therapy and Orphan Drug designations. This drug is available as an 80 mg tablet that is taken orally once daily. The double-blind, phase 3 ELATIVE trial showed that of 161 patients with PBC, 51% had a biochemical response with Iqirvo at week 52 compared to 4% of patients with placebo, for a difference of 47%; biochemical response was defined as an alkaline phosphatase (ALP) level < 1.67 times the upper limit of normal (ULN) range, with an ALP reduction of ≥ 15% from baseline, and normal total bilirubin levels.² Iqirvo is the first PPAR agonist available to treat PBC and may compete with obeticholic acid (Ocaliva®). Ipsen launched Iqirvo in June 2024 at $11,500 for a 1-month supply.

06/13/2024 Yimmugo® (immune globulin intravenous, human-dira)

The FDA has approved the IV immunoglobulin (IVIG) product Yimmugo for the treatment of primary humoral immunodeficiency (PID) in patients ≥ 2 years of age. Yimmugo is a polyvalent immunoglobulin G (IgG) formulation derived from human blood plasma and will be available as a ready-to-use solution in a single-dose vial containing 10% IgG (100 mg/mL) in 5 g/50 mL, 10 g/100 mL, and 20 g/200 mL presentations. Yimmugo is administered IV every 3 to 4 weeks. Its approval was based on data from a prospective, uncontrolled, phase 3 trial which included 67 patients and found an unadjusted serious bacterial infection (SBI) rate of 0.01 per subject-year, with only one patient experiencing an SBI, over a 12-month period.³ Yimmugo joins several other IVIG products that are available in the U.S. to treat PID. Grifols’ Biotest anticipates availability in the second half of 2024, with pricing to follow.

New biosimilars

05/28/2024 Bkemv™ (eculizumab-aeeb)

Amgen received FDA approval for Bkemv as the first interchangeable biosimilar to Alexion’s Soliris® (eculizumab). Bkemv is a complement inhibitor with indications for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiography. This product is not indicated for treatment of patients with Shiga toxin Escherichia coli-related hemolytic uremic syndrome (STEC-HUS). Bkemv is approved as a 10 mg/mL injection in a single-dose vial for IV infusion, and its dosing and boxed warnings are consistent with Soliris. This biosimilar will only be available through a Risk Evaluation and Mitigation Strategies (REMS) program. Bkemv is expected to be available in March 2025, with pricing to follow.

New indications

05/29/2024 Retevmo® (selpercatinib)

The FDA has granted Accelerated Approval to Eli Lilly’s kinase inhibitor, Retevmo, to expand its use to include pediatric patients 2 years to < 12 years of age for the following indications: (1) advanced or metastatic medullary thyroid cancer (MTC) with a rearranged during transfection (RET) mutation who require systemic therapy, (2) advanced or metastatic thyroid cancer with a RET gene fusion, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate), and (3) locally advanced or metastatic solid tumors with a RET gene fusion, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options. This approval makes Retevmo the first targeted therapy for pediatric patients < 12 years old with RET alterations. Patients should be selected based on the presence of a RET gene fusion (thyroid or other solid tumors) or specific RET gene mutation (MTC). Retevmo is dosed orally two or three times daily and should be continued until disease progression or unacceptable toxicity. These new indications received Priority Review and Orphan Drug designation. On June 12, 2024, the Accelerated Approval for the treatment of adult and pediatric patients ≥ 2 years of age with advanced or metastatic thyroid cancer with a RET gene fusion, who require systemic therapy and who are radioactive iodine-refractory, was converted to traditional approval.

05/30/2024 Breyanzi® (lisocabtagene maraleucel)

The FDA has granted a new indication to Juno Therapeutics’ CD19-directed genetically modified autologous T cell immunotherapy, Breyanzi, for the treatment of adults with relapsed or refractory mantle cell lymphoma (MCL) who have received ≥ 2 prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor. The application was reviewed under Priority Review and received Orphan Drug designation. Breyanzi is given IV as a single autologous dose that requires lymphodepleting chemotherapy prior to administration and administration in a REMS-certified health care facility. Breyanzi is the second chimeric antigen receptor (CAR) T cell therapy approved for MCL, joining brexucabtagene autoleucel (Tecartus®) by Kite.

06/10/2024 Kevzara® (sarilumab)

Sanofi’s interleukin-6 (IL-6) receptor antagonist, Kevzara, received approval of a new indication for the treatment of patients who weigh ≥ 63 kg with active polyarticular juvenile idiopathic arthritis (pJIA). Existing indications include select adults with active rheumatoid arthritis (RA) or polymyalgia rheumatica (PMR). For pJIA, Kevzara is dosed once every 2 weeks SC and can be used as monotherapy or in combination with conventional disease-modifying antirheumatic drugs (DMARDs).

06/13/2024 Augtyro™ (repotrectinib)

The FDA has granted Accelerated Approval for Bristol-Myers Squibb’s kinase inhibitor, Augtyro, for the treatment of adults and pediatric patients ≥ 12 years old with solid tumors that: (1) have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, (2) are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and (3) have progressed following treatment or have no satisfactory alternative therapy. Augtyro is dosed orally once daily for 14 days, then increased to twice daily and continued until disease progression or unacceptable toxicity. Patients should be selected for this new indication based on the presence of NTRK1/2/3 rearrangements in tumor specimens; for patients with secretory breast cancer or mammary analogue secretory cancer, consider treatment without confirmation of NTRK rearrangements in tumor specimens. The new indication was reviewed with Priority Review and was granted Breakthrough Therapy, Fast Track, and Orphan Drug designations.

06/14/2024 Blincyto® (blinatumomab)

Amgen’s bispecific CD19-directed CD3 T-cell engager, Blincyto, received approval for the new indication of treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in adult and pediatric patients ≥ 1 month of age. The application was reviewed with Priority Review and received Breakthrough Therapy and Orphan Drug designations. For the new indication, a single cycle of Blincyto monotherapy is 28 days of continuous infusion followed by a 14-day treatment-free interval for a total cycle of 42 days. For the first 3 days of the first cycle and the first 2 days of the second cycle, hospitalization is recommended. To prevent central nervous system ALL relapse, intrathecal chemotherapy prophylaxis is recommended before and during Blincyto therapy.

06/14/2024 Imfinzi® (durvalumab)

The FDA has approved a new indication for AstraZeneca’s programmed death-ligand 1 (PD-L1) blocking antibody, Imfinzi, for use in combination with carboplatin and paclitaxel followed by Imfinzi as a single agent, for the treatment of adults with primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR). It is administered IV in combination with carboplatin and paclitaxel every 3 weeks for 6 cycles followed by use as a single agent every 4 weeks.

06/17/2024 Keytruda® (pembrolizumab)

Merck’s programmed death receptor-1 (PD-1)-blocking antibody, Keytruda, has received approval for the new indication of use in combination with carboplatin and paclitaxel, followed by Keytruda as a single agent, for the treatment of adults with primary advanced or recurrent endometrial carcinoma. The application was reviewed under Priority Review. For this new indication, Keytruda is administered IV every 3 weeks or every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.

06/18/2024 Skyrizi® (risankizumab-rzaa)

The FDA has approved a new indication for Abbvie’s interleukin-23 (IL-23) antagonist, Skyrizi, for the treatment of adults with moderately to severely active ulcerative colitis (UC). This is the first IL-23 inhibitor indicated for both moderate to severe UC and moderate to severe Crohn’s disease. Following a 12-week induction period comprised of 3 doses given every 4 weeks as an IV infusion, maintenance dosing for UC is given SC every 8 weeks.

Traditional

New drugs

05/24/2024 Onyda™ XR (clonidine) oral suspension

The FDA approved Onyda XR extended-release (ER) oral suspension, by Tris Pharma, under a 505(b)(2) new drug application (NDA) for treatment of attention deficit hyperactivity disorder (ADHD) as monotherapy or as adjunctive therapy to central nervous system (CNS) stimulant medications in pediatric patients ≥ 6 years of age. This centrally acting alpha2-adrenergic agonist will be available as 0.1 mg of clonidine hydrochloride per mL of ER oral suspension for once daily nighttime administration. Onyda XR should not be substituted with other clonidine products on a mg-per-mg basis due to differences in pharmacokinetic profiles. This product is the first and only liquid nonstimulant medication for the treatment of ADHD. Onyda XR is anticipated to be available in the second half of 2024, and pricing information is forthcoming.

05/31/2024 mRESVIA® (respiratory syncytial virus [RSV] vaccine)

Moderna received FDA approval for its messenger ribonucleic acid (mRNA) vaccine, mRESVIA. This vaccine is indicated for active immunization for prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals ≥ 60 years of age. mRESVIA was granted Breakthrough Therapy designation. It will be administered as a single intramuscular (IM) dose. Approval of mRESVIA was based on results from the randomized, double-blind, placebo-controlled, phase 3 ConquerRSV study, which included 35,541 participants and found a vaccine efficacy of 83.7% against RSV-associated LRTD with ≥ 2 signs or symptoms, and 82.4% against RSV-associated LRTD with ≥ 3 signs or symptoms.4 This vaccine is the second mRNA product approved from Moderna and the only mRNA RSV vaccine. It will likely compete with the recombinant protein RSV vaccines Arexvy (RSV vaccine, adjuvanted) and Abrysvo® (RSV vaccine). The Centers for Disease Control and Prevention (CDC) has not yet indicated a preference between the available RSV vaccines for patients ≥ 60 years of age. Moderna anticipates that mRESVIA will launch in time for the 2024-2025 RSV season, with pricing to follow.

06/17/2024 Capvaxive™ (pneumococcal 21-valent conjugate vaccine)

The FDA has approved Merck’s 21-valent Capvaxive vaccine for active immunization for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 3, 6A, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15B, 15C, 16F, 17F, 19A, 20A, 22F, 23A, 23B, 24F, 31, 33F, and 35B in individuals ≥ 18 years of age. This vaccine has also been approved for active immunization for the prevention of pneumonia caused by S. pneumoniae serotypes 3, 6A, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15C, 16F, 17F, 19A, 20A, 22F, 23A, 23B, 24F, 31, 33F, and 35B in individuals ≥ 18 years of age under Accelerated Approval; continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Capvaxive was approved by Priority Review, and the CDC’s Advisory Committee on Immunization Practices (ACIP) is expected to meet in late June to make recommendations for its use in adults. This vaccine will be administered as a single-dose IM injection. Its approval was based on data from the phase 3 STRIDE trial program, including the STRIDE-3 trial, which found that Capvaxive was noninferior to pneumococcal 20-valent conjugate vaccine (PCV20) in vaccine-naïve adults, and the STRIDE-6 trial, which found that Capvaxive had comparable responses to both pneumococcal 15-valent conjugate vaccine (PCV15) and pneumococcal vaccine polyvalent (PPSV23) in vaccine-experienced adults.5,6 The safety profile of Capvaxive was also similar to its comparator vaccines in the trials. Capvaxive will likely compete with PCV20 (Prevnar 20®), which is also approved for use in adults ≥ 18 years of age. According to Merck, Capvaxive includes 8 serotypes that are not covered by the other existing pneumococcal vaccines. Availability is expected in late summer 2024 but will be determined by results of the CDC’s ACIP meeting at the end of June. Although the wholesale acquisition price is estimated at $287 per dose, patients may have access at no charge if Capvaxive is recommended by the CDC.

06/17/2024 Vigafyde™ (vigabatrin)

Pyros received FDA approval for Vigafyde, the first and only ready-to-use oral solution formulation of vigabatrin. Vigafyde is indicated as monotherapy for the treatment of infantile spasms in patients 1 month to 2 years of age for whom the potential benefits outweigh the potential risk of vision loss. The antiepileptic drug was approved under a 505(b)(2) NDA and received Orphan Drug designation. It will be available as a 100 mg/mL oral solution that does not require additional reconstitution or dilution. The product is administered twice daily. Notably, this formulation is more concentrated than other existing vigabatrin products. Because of its boxed warning for permanent vision loss, Vigafyde will only be available through a REMS program. It will likely compete with vigabatrin powder for oral solution, Sabril® (Lundbeck), and Vigpoder™ (Pyros), which can be mixed with water to form a 50 mg/mL solution for twice daily dosing. Vigafyde is anticipated to be available in the second half of 2024, with pricing to follow.

New indications

06/07/2024 Arexvy (RSV Vaccine, Adjuvanted)

Arexvy, by GlaxoSmithKline, has received an expanded indication for active immunization for the prevention of LRTD caused by RSV in individuals 50 through 59 years of age who are at an increased risk for LRTD caused by RSV. Arexvy was previously indicated only for use in individuals ≥ 60 years of age. It is administered as a single 0.5 mL IM dose.

06/07/2024 Motpoly XR™ (lacosamide)

Aucta’s antiepileptic Motpoly XR has received a new indication as adjunctive therapy in the treatment of primary generalized tonic-clonic seizures in adults and in pediatric patients weighing at ≥ 50 kg; previously, Motpoly XR was indicated for the treatment of partial-onset seizures in these patient populations. Dosing is dependent on the patient’s age as well as if the medication is being used as monotherapy or adjunctive therapy (only indicated as monotherapy for partial-onset seizures). Motpoly XR is a Schedule V controlled substance supplied as an extended-release capsule taken orally once daily.

06/12/2024 Farxiga® (dapagliflozin)

The FDA has expanded the indication for AstraZeneca’s Farxiga for use as an adjunct to diet and exercise to improve glycemic control associated with type 2 diabetes mellitus (T2DM) to include pediatric patients aged ≥ 10 years. This sodium-glucose cotransporter 2 (SGLT2) inhibitor was previously only approved for use in adults for this indication. The dosage in pediatric patients is consistent with the adult dosage for this use.

06/12/2024 Xigduo® XR (dapagliflozin/metformin)

The FDA has expanded the indication of AstraZeneca’s Xigduo XR for use as an adjunct to diet and exercise to improve glycemic control in adults with T2DM to include pediatric patients aged ≥ 10 years. Xigduo is a combination product containing dapagliflozin (an SGLT2 inhibitor) and metformin (a biguanide). It was previously approved only for use in adults for this indication. The recommended starting dosage for this indication for both adults and pediatric patients should be individualized according to the patient’s current regimen with the dosage adjusted based on tolerability and effectiveness up to the maximum daily dose (10 mg dapagliflozin/2,000 mg metformin).

First generic drug launches

Somatuline® Depot (lanreotide extended-release injection): Cipla launched its generic version of Ipsen’s Somatuline Depot prefilled syringe, a somatostatin analog indicated for (1) certain adult patients with acromegaly and (2) certain adult patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs). It is not evident if additional companies are seeking generic approval. Somatuline Depot generated $1,520 million in annual U.S. sales in 2023.

Emflaza® (deflazacort oral suspension): Tris Pharma launched its generic version of PTC Therapeutics’ Emflaza, a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients ≥ 5 years of age. It is not evident if additional companies are seeking approval for generic versions of the oral suspension.

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