Specialty Drug Pipeline Update: February 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

• Orserdu^® (elacestrant): The U.S. Food and Drug Administration (FDA) has approved Menarini Group’s Orserdu for the treatment of postmenopausal women or adult men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. Orserdu is the first oral selective estrogen receptor degrader (SERD) that has shown improved efficacy over standard of care (SOC) treatments in patients with advanced breast cancer. Orserdu was approved based on the Phase 3 EMERALD trial that demonstrated treatment with Orserdu reduced the risk of disease progression or death by 45% compared with Sandoz’s Faslodex^® (fulvestrant) or an aromatase inhibitor. The median progression-free survival (PFS) with Orserdu was 3.8 months compared to 1.9 months with the SOC.¹ Orserdu launch, and price are pending.
• Jaypirca™ (pirtobrutinib): Loxo’s Jaypirca was granted accelerated approval for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. Jaypirca, a highly selective kinase inhibitor, utilizes a novel binding mechanism and is the first FDA approved non-covalent (reversible) BTK inhibitor. The FDA’s approval is based on data from the single-arm, open-label, BRUIN Phase 1/2 trial which evaluated overall response rate (ORR) and duration of response (DOR). Jaypirca demonstrated an ORR of 50% (95% CI: 41, 59) and a median DOR of 8.3 months (95% CI: 5.7, not estimable).² Loxo is planning on launching Jaypirca in the next few weeks with pricing to follow.
• Jesduvroq™ (daprodustat): The FDA approved GlaxoSmithKline’s Jesduvroq as the first oral treatment for anemia caused by chronic kidney disease (CKD) for adults who have been receiving dialysis for at least four months. Safety has not been established for patients who are not on dialysis. Jesduvroq increases erythropoietin levels which are low in people with CKD who require dialysis. Jesduvroq was analyzed in the ASCEND Phase 3 clinical program, which included 5 trials and met its primary endpoints in assessing the efficacy and safety of daprodustat for the treatment of anemia caused by CKD in adults that require dialysis compared to injected recombinant human erythropoietin. Jesduvroq raised and maintained the hemoglobin within the target range of 10-11 grams/deciliter, similar to that of the recombinant human erythropoietin.³In November, the FDA Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 13-3 that the advantage of treatment with Jesduvroq outweighs its risks for use in adult dialysis patients with anemia of CKD. However, among adult non-dialysis patients with anemia of CKD, CRDAC voted 11-5 that the benefit of treatment with daprodustat does not outweigh the risks.⁴ Jesduvroq launch and price are pending.
• Syfovre™ (pegcetacoplan injection): The FDA has approved Apellis Pharmaceuticals’ Syfovre for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Syfovre is the first FDA-approved treatment for GA, a leading cause of blindness that impacts more than one million people in the U.S. and five million people worldwide. The FDA based its approval on results from Apellis’ late-stage DERBY and OAKS studies. The trials found Syfovre lowered the rate of GA lesion growth compared to placebo and demonstrated increased treatment effects over time. The greatest benefit seen at a 36% reduction in lesion growth between the 18- and 24-month mark.⁵ Syfovre is expected to release in the beginning of March with a cost of $2,190 per vial.
• Filspari™ (sparsentan): The FDA has granted accelerated approval to Travere Therapeutics’ Filspari to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g. Filspari is an endothelin and angiotensin II receptor antagonist. The rare disease is characterized by the accumulation of IgA in the kidneys, which causes cellular changes within the glomeruli and damages the glomerular filtration barrier. The accelerated approval was based on clinical trial data indicating a reduction of proteinuria; however, it has not been established whether Filspari slows kidney function decline in patients with IgAN. Results from the Phase 3 PROTECT study, showed that patients treated with Filspari achieved a mean reduction in proteinuria of 45% compared to 15.1% with irbesartan after 36 weeks of treatment.⁶ Filspari is planning to launch in late February with pricing to follow.
• Lamzede™ (velmanase alfa-tycv): Chiesi Global Rare Diseases’ Lamzede has been approved by the FDA for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme α-mannosidase. The prevalence of AM is approximately one in every 500,000 to one in every 1,000,000 babies born worldwide. The approval was based on efficacy and safety data from multiple clinical trials including a Phase 3 study that compared Lamzede against placebo. Treatment with Lamzede demonstrated improvements in the 3-minute stair climbing test, 6-minute walking test, and forced vital capacity at 12 months. Moreover, the efficacy of Lamzede was supported by a reduction in serum oligosaccharide concentration.⁷ Lamzede is supplied in a single-dose vial containing 10mg of velmanase alfa as lyophilized powder for intravenous infusion after reconstitution. Launch and price are pending.

New Indications

• Trodelvy^® (sacituzumab govitecan-hziy): The FDA expanded the indication of Gilead Sciences’ Trodelvy to include the treatment of adult patients with unresectable locally advanced or metastatic ER+, HER2-, (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting.
• Keytruda^® (pembrolizumab): Merck’s Keytruda has been granted a new indication by the FDA for adjuvant treatment of patients with stage IB, II or IIIA non-small cell lung cancer (NSCLC) following complete surgical resection.
• Tukysa^® (tucatinib): The FDA expanded the indication of Seagen’s Tukysa for treatment of adults with HER2-positive colorectal cancer who have received at least one prior treatment regimen for unresectable or metastatic disease.
• Brukinsa^® (zanubrutinib): The FDA granted approval a new indication BeiGene’s Brukinsa to include the treatment of adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

February News

• “Amgen Inc said it launched a biosimilar version of AbbVie Inc’s big selling arthritis treatment, the first such competition for Humira in the United States. Amgen said its drug, Amjevita, or adalimumab-atto, will have two list prices – $3,288 and $1,557 per 40 milligram pen device for a two-week supply – representing a discount of 5% and 55% to Humira, depending on who is purchasing.”⁸
• “Intercept Pharmaceuticals, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted Intercept’s New Drug Application (NDA) for obeticholic acid (OCA) seeking accelerated approval for the treatment of patients with pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis (NASH). FDA indicated that it considers this a complete, Class 2 resubmission and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 22, 2023, for the NDA. The timeline for the review of the NDA by FDA remains subject to change.”⁹
• “The Global Biobetters Marketis poised to value $89,957 million by 2028 end at a CAGR of 8.3% during the forecast period 2022 to 2028. Global biobetters have been attracting increasing interest from drug manufacturers. They are a way to increase efficacy and decrease risk, especially in the area of immunogenicity. Biobetters are made using existing peptides, or protein-based therapies. These key parameters are modified during the process to alter stability, affinity, or selectivity. The low risk of commercial failure is probably the main reason for increased investments in biobetters. A favorable factor for biopharmaceutical firms is the low cost associated with research and development when developing biologics. Research has improved their understanding of regulatory requirements to establish bioequivalence between reference products and comparator products. This knowledge has allowed them to develop biosetters of high stability. Biobetters have seen rapid development thanks to the design of protein-based methods. Biobetters have been a strong competitor to biosimilars because of the need to improve safety and efficacy.”¹⁰

References

1. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-elacestrant-er-positive-her2-negative-esr1-mutated-advanced-or-metastatic-breast-cancer
2. https://investor.lilly.com/news-releases/news-release-details/us-fda-approves-jaypircatm-pirtobrutinib-first-and-only-non
3. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-treatment-anemia-caused-chronic-kidney-disease-adults-dialysis/
4. https://www.ajmc.com/view/fda-approves-daprodustat-for-anemia-from-ckd-in-adult-dialysis-patients
5. https://www.biospace.com/article/fda-approves-apellis-syfovre-as-first-treatment-for-geographic-atrophy/
6. https://ir.travere.com/news-releases/news-release-details/travere-therapeutics-announces-fda-accelerated-approval
7. https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-announces-fda-approval-of-lamzedevelmanase-alfa-tycv-for-alpha-mannosidosis-301749440.html?tc=eml_cleartime
8. https://www.reuters.com/business/healthcare-pharmaceuticals/amgen-launches-biosimilar-version-abbvies-humira-2023-01-31/
9. https://ir.interceptpharma.com/news-releases/news-release-details/fda-accepts-intercepts-new-drug-application-oca-treatment-pre
10. https://www.marketwatch.com/press-release/biobetters-market-report-trends-growth-size-share-and-forecast-2028-2023-01-17