Traditional Drug Approvals Monthly Update: December 2023

This monthly approvals wrap-up provides a review of newly approved traditional drugs, recent drug launches, generic updates, new indications and news of note on drugs in the approval process.

See separate article for approval information on specialty drugs.

New Drug Information

Fabhalta® (iptacopan): Under a Priority Review, the United States (U.S.) Food and Drug Administration (FDA) approved Fabhalta for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). The complement factor B inhibitor by Novartis is designed to regulate intra- and extravascular hemolysis. It is the first oral monotherapy for the treatment of PNH and the oral capsule is dosed twice daily. The open-label, phase III APPLY-PNH (NCT04558918) trial evaluated Fabhalta in 97 patients with residual anemia (hemoglobin < 10 g/dL) despite prior anti-C5 treatment (eculizumab or ravulizumab).¹ At week 24, 82.3% of patients who switched to Fabhalta experienced a sustained increase in hemoglobin (Hb) levels ≥ 2 g/dL from baseline in the absence of transfusions (co-primary endpoint) compared to 0% who continued on anti-C5 treatment (p<0.0001). In addition, 67.7% of patients who switched to Fabhalta had a sustained Hb ≥ 12 g/dL without the need for transfusions (co-primary endpoint) compared to 0% who continued anti-C5 treatment (p<0.0001). Labeling for Fabhalta includes a boxed warning for serious infections caused by encapsulated bacteria and should not be used in patients with this type of infection. Fabhalta is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Fabhalta was granted Breakthrough Therapy and Orphan Drug designations by the FDA. Fabhalta is available through the specialty pharmacy, Onco360®.² Fabhalta has launched with an annual wholesale acquisition cost of $550,000.

iDose® TR (travoprost intracameral implant): The FDA approved a 505(b)(2) new drug application (NDA) for iDose TR, by Glaukos. It is indicated to reduce intraocular pressure (IOP) in patients with ocular hypertension (OHT) or open-angle glaucoma (OAG). iDose TR is approved as a 75 mcg intracameral implant for use as a single administration per eye. In two phase 3 trials, iDose TR demonstrated non-inferiority to timolol 0.5% topical ophthalmic solution (instilled into the eye twice a day), based on IOP lowering from baseline to 3 months (range, 6.6-8.4 mm Hg versus 6.5-7.7 mm Hg, respectively). However, non-inferiority was not seen over the next 9 months.³ In addition, at 12 months, 81% of patients in the iDose TR arm were completely free of IOP-lowering topical medications in both trials. IOP-lowering and a marked decrease in topical IOP-lowering medication burden was seen for up to 36 months following administration of a single iDose TR implant. iDose TR is the first intracameral implant that allows for continuous delivery of medication for an extended period of time to treat OHT or OAG. In doing so, it may improve patient adherence and side effects associated with topical agents. Launch of iDose TR is planned in Q1 of 2024. Pricing will follow.

Filsuvez® (birch triterpenes): The FDA approved Filsuvez, by Chiesi Global Rare Diseases, for the treatment of partial thickness wounds in patients ≥ 6 months of age with junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB). Epidermolysis bullosa (EB) is a rare, genetic disorder that is characterized by painful blisters that develop on the skin or mucous membranes as a result of mechanical trauma or friction. Filsuvez was granted Fast Track, Orphan Drug and Rare Pediatric Disease designations during its development for EB. Filsuvez is approved as a topical gel that contains naturally occurring substances derived from birch bark known as triterpenes. The gel is self-administered and is applied to EB wounds at each dressing change (every 1 to 4 days). In the double-blind, EASE (NCT03068780; n=223) trial, 41.3% of patients with JEB or DEB treated with Filsuvez achieved the primary endpoint of first complete closure of target wound by day 45 compared to 28.9% who received placebo; response rates of 50.5% and 43.9%, respectively, were reported through day 90.⁴ Filsuvez is the first treatment approved for JEB and the second treatment approved for DEB, following approval of the topical gene therapy Vyjuvek® (beremagene geperpavec). Launch timeframe for Filsuvez in the U.S. has not been announced; pricing will follow.

New Indications

Cresemba® (isavuconazonium): Cresemba, an azole antifungal by Astellas, received expanded indications to include pediatric patients for the treatment of invasive aspergillosis and invasive mucor-mycosis; previously it was only approved for use in adults. Notably, the 372 mg injection is approved for use in patients ≥ 1 year of age and the 74.5 mg and 186 mg capsules are approved for use patients ≥ 6 years of age who weigh ≥ 16 kg. The 74.5 mg is a new capsule strength to accommodate pediatric dosing. Recommended dosages are based on the patient age and body weight.⁵

Nexletol® (bempedoic acid) and Nexlizet® (bempedoic acid/ezetimibe): Esperion’s lipid-lowering agents, Nexletol and Nexlizet, received an updated indication to include the treatment of primary hyperlipidemia as a qualifier for existing approved populations. In addition, the maximally tolerated qualifier for statin use has been removed. The indication now reads, as an adjunct to diet and statin therapy for the treatment of primary hyperlipidemia in adults with heterozygous familial hypercholesterolemia (HeFH) or atherosclerotic cardiovascular disease, who require additional lowering of LDL-C.^6,7

Zoryve® (roflumilast 0.3% foam): Arcutis Biotherapeutics received an expanded indication for Zoryve to include pediatric patients ≥ 9 years of age for the topical treatment of seborrheic dermatitis. Previously, it was only approved for use in adults.⁸

Generic Drug Information

Condylox® (podofilox gel): Padagis launched its generic version of Allergan’s Condylox gel for the topical treatment of anogenital warts. Condylox generated $11 million in U.S. annual sales in 2022.

Forteo® (teriparatide): Apotex launched their generic version and Prasco launched an authorized generic version of Eli Lilly’s Forteo for the treatment of osteoporosis in select postmenopausal women, osteoporosis associated with sustained systemic glucocorticoid therapy in men and women, and to increase bone mass in select men with primary or hypogonadal osteoporosis. Teva’s generic version is expected to be available in the coming weeks. Forteo generated $723 million in U.S. annual sales in 2022.

Risperdal Consta® (risperidone microspheres): Teva Pharmaceuticals launched their generic version of Janssen’s Risperdal Consta for the treatment of bipolar disorder and schizophrenia. Viatris and Lupin are expected to launch their generic versions in 2025 and 2026, respectively. Risperdal Consta generated $334 million in U.S. annual sales in 2022.