Drug Approvals Monthly Update: March 2024

Critical updates in an ever-changing environment

March 27, 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

3/13/2024 Tevimbra™ (tislelizumab-jsgr)

Tevimbra, a programmed death receptor-1 (PD-1) blocking antibody, was approved by the FDA for the treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(Ligand)1 inhibitor. Tevimbra is administered by intravenous (IV) infusion every three weeks. In the open-label, phase 3 RATIONALE-302 trial, Tevimbra led to a significantly longer overall survival (OS), the primary endpoint, compared to investigator’s choice of chemotherapy (median OS, 8.6 versus 6.3 months, respectively; hazard ratio [HR], 0.7 [95% confidence interval [CI], 0.57 to 0.85]; p=0.0001).¹ Survival benefit was consistent in patients with programmed death-ligand 1 tumor area positivity (TAP) score ≥ 10% (HR, 0.54). Tevimbra received Orphan Drug designation from the FDA. It will compete with the PD-1/PD-L1 inhibitors Keytruda® (pembrolizumab) and Opdivo® (nivolumab) in the ESCC space. Beigene plans to launch Tevimbra in the U.S. in 1H2024, with pricing to follow.

3/18/2024 Lenmeldy™ (atidarsagene autotemcel)

The FDA approved Lenmeldy, by Orchard Therapeutics, for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). This is the first treatment approved for MLD, a rare and fatal genetic lysosomal storage disorder that affects the brain and nervous system causing progressive loss of motor and cognitive function. Lenmeldy was granted a Priority Review as well as Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy (RMAT) designations. The gene therapy is manufactured from the patient’s own hematopoietic stem cells (HSCs), which are genetically modified to include functional copies of the arylsulfatase A (ARSA) gene. The engineered cells (Lenmeldy) are transplanted back into the patient by a one-time single-dose IV infusion at a qualified treatment center. Treatment also requires myeloablation of the bone marrow and conditioning prior to the Lenmeldy infusion. Safety and efficacy of Lenmeldy were evaluated based on data from 37 patients in two phase 1/2 open-label, single-arm studies and one expanded access framework compared to the natural course of disease in 49 patients.² Patients treated with Lenmeldy experienced statistically significant and clinically meaningful improvement in severe motor impairment-free survival (primary endpoint) compared to natural course of disease. In addition, Lenmeldy significantly extended overall survival (in patients with PSLI MLD) and preserved cognitive abilities. Five qualified treatment centers are in the process of becoming fully qualified to administer Lenmeldy. Orchard announced a wholesale acquisition cost (WAC) of $4.25 million for the one-time treatment.

3/19/2024 Tryvio™ (aprocitentan)

Idorsia received FDA approval for Tryvio for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adults who are not adequately controlled on other drugs. Tryvio is the first dual endothelin receptor antagonist to treat resistant hypertension, Tryvio oral tablet is administered once daily. In phase 3 PRECESION trial, a significant reduction in the primary endpoint of least square (LS) mean change in office systolic blood pressure (SBP) at week 4 was demonstrated with Tryvio 12.5 mg and 25 mg doses compared to placebo (placebo-adjusted change, -3.8 mm Hg and -3.7 mm Hg, respectively; p<0.005 for both) when added to background antihypertension therapy. A reduction in office diastolic blood pressure (DBP) (secondary endpoint) was also seen with each dose compared to placebo (placebo-adjusted change, -3.9 mm Hg and -4.5 mm Hg, respectively). SBP and DBP reductions seen with aprocitentan were maintained for 32 weeks. Launch of Tryvio is expected in 2H 2024, with pricing to follow.

New biosimilars

2/23/2024 Simlandi® (adalimumab-ryvk)

Alvotech announced FDA approval of Simlandi, its interchangeable biosimilar to Abbvie’s tumor necrosis factor (TNF) blocker Humira® (adalimumab). Simlandi holds all the same indications as the reference produce, which include rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), plaque psoriasis (PSO), psoriatic arthritis (PsA), ankylosing spondylitis (AS), Crohn’s disease (CD), ulcerative colitis (UC), hidradenitis suppurativa (HS) and uveitis (UV), as well as the boxed warnings for serious infections and malignancy. It is the first high-concentration, citrate-free, interchangeable biosimilar to Humira and will be available as a 40 mg/0.4 mL single-dose autoinjector in 1- and 2-count cartons. Teva will begin to commercialize Simlandi in the U.S. in the immediate future, with pricing to follow.

3/05/2024 Jubbonti® (denosumab-bbdz)

The FDA approved denosumab-bbdz as brand name Jubbonti, an interchangeable biosimilar to Amgen’s Prolia (denosumab), a RANK ligand (RANKL) inhibitor indicated for the treatment of osteoporosis in postmenopausal women and glucocorticoid-induced osteoporosis in men and women, and to increase bone mass in men with osteoporosis or receiving androgen deprivation therapy, or in women receiving aromatase inhibitor therapy. Jubbonti is the first biosimilar to Prolia. Like its reference product, Jubbonti carries a boxed warning for severe hypocalcemia in patients with advanced kidney disease. Jubbonti is approved as 60 mg/1 mL solution for subcutaneous (SC) administration. The launch timeframe of Jubbonti will depend on ongoing litigation. Pricing has not been announced.

3/05/2024 Wyost® (denosumab-bbdz)

The FDA also approved denosumab-bbdz as brand name Wyost as an interchangeable biosimilar to Amgen’s Xgeva® (denosumab), which is indicated for the prevention of skeletal-related events in select patients with multiple myeloma and in certain patients with bone metastases from solid tumor, giant cell tumor of bone or hypercalcemia of malignancy. Wyost is approved as 120 mg/1.7 mL solution for SC administration. Wyost is the first biosimilar to Xgeva. The launch timeframe of Wyost will depend on ongoing litigation. Pricing has not been announced.

3/05/2024 Tyenne® (tocilizumab-aazg)

Fresenius Kabi announced the approval of Tyenne, a biosimilar to IV and SC formulations of Actemra® (tocilizumab). Tyenne is indicated for the treatment of RA, giant cell arteritis, polyarticular JIA, and systemic JIA. Tyenne is not interchangeable to Actemra and is not approved for systemic sclerosis-associated interstitial lung disease, cytokine release syndrome or Coronavirus disease 2019 (COVID-19). Tyenne is approved as a 20 mg/mL solution for IV infusion and a 162 mg/0.9 mL solution in a prefilled syringe and autoinjector. The specific launch date in the U.S. has not been announced; pricing will follow.

New indications

2/28/2024 Ziextenzo® pegfilgrastim-bmez

Sandoz’ leukocyte growth factor Ziextenzo, a biosimilar to Amgen’s Neulasta®, received a new indication to increase survival in patients acutely exposed to myelosuppressive doses of radiation (hematopoietic subsyndrome of acute radiation syndrome [H-ARS]). Ziextenzo is given as two doses administered SC one week apart for H-ARS. It now carries all indications as its reference product.

3/01/2024 Rybrevant® (amivantamab-vmjw)

The FDA approved Rybrevant, a bispecific EGF receptor-directed and MET receptor-directed antibody by Janssen, for use in combination with chemotherapy (carboplatin-pemetrexed) for the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. This is the first agent approved for first-line treatment in this patient population.

3/06/2024 Besponsa® (inotuzumab ozogamicin)

The FDA expanded the indication for Pfizer’s CD22-directed antibody and cytotoxic drug conjugate, Besponsa, to include pediatric patients 1 year and older for the treatment of relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). Previously, Besponsa was only approved for use in adults.

3/06/2024 Opdivo® (nivolumab)

The FDA approved Opdivo, a PD-1 blocking antibody by Bristol-Myers Squibb, for use in combination with cisplatin and gemcitabine for the first-line treatment of adults with unresectable or metastatic urothelial carcinoma.

3/07/2024 Brukinsa® (zanubrutinib)

The FDA granted Accelerated Approval to Beigene’s Brukinsa for the treatment of adults with relapsed or refractory (R/R) follicular lymphoma (FL), for use in combination with obinutuzumab, after two or more lines of systemic therapy. Continued approval for this indication may depend on results from a confirmatory trial. Brukinsa is the first Bruton’s kinase (BTK) inhibitor indicated for R/R FL in the U.S. This indication received Fast Track and Orphan Drug designations.

3/13/2024 Livmarli® (maralixibat)

Mirum Pharmaceuticals received a new indication for Livmarli for the treatment of cholestatic pruritus in patients ≥ 5 years of age with progressive familial intrahepatic cholestasis (PFIC). The ileal bile acid transporter (IBAT) inhibitor received Orphan Designation for PFIC and Breakthrough Therapy designation for PFIC type 2.

3/14/2024 Breyanzi® (lisocabtagene maraleucel)

Bristol-Myers Squibb announced that they received Accelerated Approval for their CD19-directed chimeric antigen receptor (CAR) T-cell therapy, Breyanzi, for the treatment of adults with R/R chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. Continued approval may depend on results from confirmatory trials. Breyanzi was granted a Priority Review and an Orphan Drug designation for this indication and is the first CAR T approved for R/R CLL/SLL.

3/15/2024 Edurant® and Edurant® PED (rilpivirine)

Janssen received and expanded indication for Edurant for its use in combination with other antiretroviral agents to include the treatment of human immunodeficiency virus (HIV)-1 infected, treatment-naïve pediatric patients with HIV-1 RNA ≤ 100,000 copies/mL, who are 2 years to < 12 years of age and weigh ≥ 25 kg to < 35 kg. Previously it was only approved for use in patients ≥ 12 years of age weighing ≥ 35 kg. To accommodate oral administration in the new younger population, the FDA approved Edurant PED 2.5 mg tablets for oral suspension.

3/19/2024 Iclusig® (ponatinib)

Takeda’s tyrosine kinase inhibitor, Iclusig, gained a new indication for the treatment of adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), for use in combination with chemotherapy; this indication is approved under Accelerated Approval, and continued approval may depend upon verification of clinical benefit in a confirmatory trials. Iclusig was also granted standard approval as monotherapy for Ph+ ALL in adults for whom no other kinase inhibitors are indicated or with T315I-positive Ph+ ALL. Iclusig received a Priority Review and Orphan Drug designation for Ph+ ALL.

3/18/2024 Spevigo® (spesolimab-sbzo)

Spevigo, an interleukin-36 receptor antagonist by Boehringer Ingelheim, received an expanded indication for IV administration for the treatment of generalized pustular psoriasis (GPP) flares to include pediatric patients ≥12 years of age weighing ≥ 40 kg; previously, it was only approved for use in adults for GPP flares. Spevigo also gained a new indication for SC administration for maintenance therapy of GPP (when not experiencing a flare) in adults and pediatric patients ≥ 12 years of age weighing ≥ 40 kg. The recommended SC maintenance dosage is a 600 mg loading dose (four 150 mg injections), if required, that is administered by a health care professional. Subsequent doses of 300 mg (two 150 mg injections) may be self- or caregiver- administered SC 4 weeks later and every 4 weeks thereafter. A 150 mg/mL solution in a single-dose prefilled syringe was FDA approved for SC maintenance dosing.

Traditional

New drugs

3/04/2024 clobetasol propionate 0.05%

The FDA approved the first clobetasol propionate ophthalmic formulations. The 0.05% suspension by Formosa is indicated for the treatment of post-operative inflammation and pain following ocular surgery. It is administered twice daily for 2 weeks beginning the day after surgery.³ This product was approved under the FDA’s 505(b)(2) approval pathway allowing for use of data not developed by the drug applicant. Launch and pricing information have not been announced.

3/14/2024 Rezdiffra™ (resmetirom)

The FDA granted an Accelerated Approval for Madriga’s once-daily, oral thyroid hormone receptor-beta (THR-beta) agonist, Rezdiffra, for use in conjunction with diet and exercise for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). The FDA has granted Rezdiffra Breakthrough Therapy and Fast Track designations as well as a Priority Review, but continued approval may depend on verification and description of clinical benefit in confirmatory trials. Rezdiffra treats the underlying cause of NASH by decreasing liver fat, thereby reducing lipotoxicity and inducing NASH resolution. In the double-blind, phase 3 MAESTRO-NASH trial, at 52 weeks, Rezdiffra (80 mg and 100 mg orally once daily) led to resolution of NASH (25.9% and 29.9% of patients, respectively, versus 9.7% with placebo; p<0.001 for both doses) and a reduction in liver fibrosis (24.2% and 25.9%, respectively, versus 14.2% with placebo; p≤0.001 for both doses).4 It is the first medication  approved by the FDA to treat NASH and is expected to be available in the U.S. in April 2024 through a limited specialty pharmacy network. The anticipated annual cost of Rezdiffra is $47,400.

New indications

2/23/2024 Biktarvy® (bictegravir/emtricitabine/tenofovir alafenamide)

Biktarvy, by Gilead, received an expanded indication as a complete regimen to treat adult and pediatric patients weighing at least 14 kg with HIV-1 who have suppressed viral loads (HIV-1 RNA < 50 copies per mL) with known or suspected M184V/I resistance, which is a common type of treatment resistance.

2/28/2024 Veklury® (remdesivir)

The FDA expanded the indication for Gilead’s severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) nucleotide analog RNA polymerase inhibitor, Veklury, for the treatment of COVID-19 to include pediatric patients from birth to < 28 days of age weighing ≥ 1.5 kg to < 3 kg. The recommended dosage in pediatric patients is based on the patient’s weight, as described in the prescribing information, with an IV loading dose given on day 1, followed by daily doses for a total of 5 or 10 days, depending on patient clinical status.

3/08/2024 Praluent® (alirocumab)

The FDA has expanded the indication for Praluent, a proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor by Regeneron, to reduce low-density lipoprotein cholesterol (LDL-C) in pediatric patients ≥ 8 years of age with heterozygous familial hypercholesterolemia (HeFH), when used as an adjunct to diet and other LDL-C-lowering therapies. Praluent is dosed SC once every 4 weeks as 150 mg in pediatric patients weighing < 50 kg and 300 mg in those ≥ 50 kg.

3/08/2024 Wegovy® (semaglutide)

FDA approved Wegovy to reduce the risks of major adverse cardiovascular events (MACE) including cardiovascular (CV) death, non-fatal myocardial infarction (MI) or non-fatal stroke in adults with either overweight or obesity and established cardiovascular disease (CVD). The glucagon-like peptide-1 (GLP-1) receptor agonist by Novo Nordisk is also indicated for long-term weight reduction among those who are obese or overweight with a weight-related comorbid condition.

03/15/2024 Xhance® (fluticasone propionate) nasal spray

Xhance nasal spray, by Optinose, was FDA approved for chronic rhinosinusitis without nasal polyps (CRSsNP) in adults. Xhance is a drug-device system that uses the Exhalation Delivery System™ to deliver a twice daily dose of the steroid fluticasone propionate into the nasal cavity. It is the first agent for treatment of adults with CRSsNP. Xhance is also indicated for chronic rhinosinusitis with nasal polyps (CRSwNP).

First generic drug launches

Thiola EC® (tiopronin delayed release) tablets 100 mg, 300 mg): Torrent launched their generic version of Thiola EC tablets for use in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in select patients with severe homozygous cystinuria. Thiola EC generated < $10 million in annual U.S. sales in 2023.

Rectiv™ (nitroglycerin) ointment 0.4%: Cosette launched its generic version of Rectiv, for the treatment of moderate to severe pain associated with chronic anal fissure. Rectiv generated $23 million in annual U.S. sales in 2023.

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