Specialty Drug Pipeline Monthly Update: August 2023

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

See separate article for pipeline information on traditional drugs.

New Drug Information

• Vanflyta^® (quizartinib): The U.S. Food and Drug Administration (FDA) granted accelerated approval to Daiichi Sankyo’s Vanflyta (quizartinib) oral tablets for the treatment of FLT3-ITD positive acute myeloid leukemia (AML) who are newly diagnosed in combinations with cytarabine and anthracycline for induction and cytarabine consolidation and monotherapy maintenance in patients without transplant. The QuANTUM-First trial demonstrated that Vanflyta in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation, and continued as maintenance monotherapy, results in a 22% reduction in the risk of death compared to chemotherapy alone (p=0.032). The complete remission rates were similar between the two treatment groups. The median duration of CR was 38.6 months in the Vanflyta group compared to those receiving chemotherapy alone (p<0.05).¹ Vanflyta has launched with a list price of $546 per tablet for both strengths (17.7 and 26.5mg).
• Izervay^® (avacincaptad pegol): Astellas/Iveric Bio’s Izervay (avacincaptad pegol) for intraocular injection was FDA approved for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). In two Phase 3 trials, GATHER1 and GATHER2, the administration of Izervay given once monthly over 12 months showed a statistically significant reduction in the rate of GA growth compared to those that were given a placebo.² Izervay will launch at a price of $2,100 per vial. Apellis’ Syfovre, also approved for GA secondary to AMD, is priced at $2,190 and can be administered once every 25 to 60 days.
• Talvey™ (taquetamab-tgvs): Johnson and Johnson (J & J) received accelerated approval of Talvey, an off-the-shelf bispecific G protein-coupled receptor family C group 5 member D (GPRC5D)-directed CD3 T-cell engager for subcutaneous injection, for the treatment of relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Patients must be hospitalized for 48 hours after all doses within the step-up dosing schedule due to potential cytokine release syndrome. The Phase 2 MonumenTAL-1 study showed meaningful overall response rates (ORR) and durability of response (DUR). This indication is contingent upon a clinical benefit demonstrated in confirmatory trial.³ The list price has been set at ~ $45,000 per month. J & J’s Tecvayli which is also a bispecific antibody with a similar indication is priced ~$39,500 per month. Both use weight-based dosing and after step-up dosing are administered weekly.
• Elrexfio™ (elranatamab-bcmm): Pfizer also received accelerated approval for their off the shelf bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3 formulated for subcutaneous delivery for treatment of relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunodulatory agent, and an anti-CD38 monoclonal antibody. Patients must be hospitalized for 48 hours after administration of the first step-up dose, and for 24 hours after administration of the second step-up dose. The MagnetisMM-3 open-label, single-arm, multi-center study showed meaningful response rates and DUR. This indication is contingent upon a clinical benefit demonstrated in confirmatory trials.⁴ The list price is set at $41,500 per month. After step-up dosing, a fixed-dose of Elrexfio is administered weekly through week 24. Depending on the patient’s response, the dose interval could transition to an every two-week schedule.
• Akeega™ (niraparib and abiraterone acetate): J & J’s combination oral tablet, Akeega, was approved in combination with prednisone for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC). About 10-15% of patients with mCRPC are BRCA-positive. Safety and efficacy was demonstrated in the Phase 3 MAGNITUDE trial which demonstrated a reduced risk of radiographic disease progression or death by 47% in the indicated patient population who received Akeega plus prednisone compared to those who received placebo plus abiraterone acetate plus prednisone.⁵ The list price for a 30-day supply is $18,750 and will be available in mid-September.
• Sohonos™ (palovarotene): Ipsen received FDA approval of Sohonos, a once daily oral medication, for the reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressiva (FOP) which affects about 400 people in the U.S. Study results from the Phase 3 MOVE trail demonstrated that patients treated with palovarotene had reduced annualized heterotopic ossification volume compared with no treatment. Sohonos causes premature epiphyseal closure in growing pediatric patients; close monitoring is recommended.⁶ Sohonos is the first FDA approved medication for FOP. The average annual list price is $624,000.
• Veopoz™ (pozelimab-bbfg): The FDA granted approval for Regeneron’s Veopoz, the first treatment FDA approved for CD55-deficient protein-losing enteropathy, CHAPLE disease, in adult and pediatric patients 1 year of age and older. CHAPLE is a rare disease with fewer than 10 patients diagnosed in the U.S. In one single-arm study, 10 people with CHAPLE received a weight-based IV administered loading dose, followed by a weekly subcutaneous injection; both formulations must be prepared and administered by a health care provider. All patients achieved a serum albumin concentration of at least 3.5 g/dL by week 12 which was maintained through at least Week 72. All patients demonstrated a reduction in the number of hospitalizations and number of albumin transfusions over 48 weeks compared to prior treatment. The list price is $34,615 per single-use vial and will be available during the third quarter of 2023.^7-8
• Eylea^® HD (afibercept): After the FDA declined to approve Regeneron’s Eylea HD in June due to manufacturing issues, it was approved in August. Like Eylea, Eylea HD is approved for wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. Eylea is also approved for retinopathy of prematurity and macular edema following retinal vein occlusion. Eylea is available as 2mg intravitreal injections that are administered as maintenance dosing every 4 to 12 weeks for wet AMD and every 4 to 8 weeks for DME and DR. Eylea is available as an 8 mg injection given as maintenance dosing every 8 to 16 weeks for wet AMD, every 8 to 16 weeks for DME, and every 8 to 12 weeks for DR.⁹ The list price is $2,625 per single-use vial Eylea HD will also compete with market share with Eylea biosimilars and Roche’s Vabysmo^® (faricimab-svoa).
• Tyruko^® (natalizumab-sztn): Sandoz received FDA approval for their biosimilar of Biogen’s Tysabri^® (natalizumab, intravenous formulation) for the treatment of multiple sclerosis and Crohn’s disease. Due to patent litigation, the launch timing of Tyruko is not certain. Sandoz could launch ‘at risk’ while the court case is ongoing.¹⁰
• Hepzato Kit™ [melphalan/Hepatic Delivery System (HDS)]: Delcath Systems received FDA approval of the Hepzato Kit for adult patients with metastatic uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation. Hepzato was approved via the 505(b)(2) pathway with the reference product being Hepzato^® (melphalan). The Hepzato Kit allows for percutaneous hepatic perfusion which permits higher melphalan exposure in target tissue and lower systemic toxicity.¹¹ Launch is anticipated in fourth quarter of 2023 with pricing to follow.

 New Indications

• Lonsurf ^® (tipiracil HCl/trifluridine): The FDA expanded the label of Taiho’s Lonsurf to include use in combination with bevacizumab, for metastatic colorectal cancer (mCRC) previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type, an anti-EGFR therapy.

References

1. FDA approves quizartinib for newly diagnosed acute myeloid leukemia | FDA
2. Iveric Bio Receives FDA Approval for Izervay for Geographic Atrophy (eyewire.news)
3. J&J wins FDA nod for novel multiple myeloma drug Talvey (fiercepharma.com)
4. FDA approves bispecific drug from Pfizer for multiple myeloma | BioPharma Dive
5. https://www.formularywatch.com/view/fda-approves-combination-tablet-for-metastatic-prostate-cancer
6. https://www.biopharmadive.com/news/ipsen-fda-sohonos-fibrodysplasia-ossificans-progressiva/691151/#:~:text=It’s%20designed%20to%20reduce%20the,patient%20needs%20and%20support%20programs.
7. US FDA approves Regeneron’s ultra-rare blood disease drug (msn.com)
8. fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-cd55-deficient-protein-losing-enteropathy-chaple-disease
9. Regeneron’s Eylea could return to growth after nod to high-dose version- analysts | Reuters
10. FDA approves Novartis’ copycat of blockbuster Biogen drug | BioPharma Dive
11. https://www.prnewswire.com/news-releases/delcath-systems-inc-announces-fda-approval-of-hepzato-kit-for-the-treatment-of-adult-patients-with-unresectable-hepatic-dominant-metastatic-uveal-melanoma-301900346.html