Specialty Drug Pipeline Update: April 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

• Zynyz^®(retifanlimab-dlwr): The U.S. Food and Drug Administration (FDA) has granted accelerated approval of Incyte’s Zynyz, a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1), for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). MCC is a rare and aggressive type of skin cancer that tends to grow quickly and has a high rate of metastatic disease, leading to a poor prognosis. The estimated five-year overall survival (OS) rate is 14% in patients with MCC who present with distant metastatic disease. MCC impacts less than 1 per 100,000 people in the U.S. Zynyz was approved based on the clinical program POD1UM-201 trial which demonstrated among chemotherapy-naïve patients, Zynyz monotherapy resulted in an objective response rate (ORR) of 52%.¹ Complete response was seen in 12 patients (18%), and 22 patients (34%) showed partial response. Among the responding patients, the duration of response (DOR) ranged from 1.1 to 24.9+ months, and 76% experienced a DOR of six months or longer, and 62% experienced a DOR of 12 months. Zynyz monthly price will be “comparable” to other agents in the class that are on the market, a spokesperson from Incyte said. Keytruda has a list price of $10,683 for each dose when given every three weeks and $21,367 for each dose when given every six weeks. Zynyz’s launch and price are pending.
• Joenja™ (leniolisib): The FDA has approved Pharming’s Joenja for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older.  APDS is a rareprimary immunodeficiency disease affecting one to two people per million. Joenja was approved based on the Study 2201, a Phase 2/3 study of 31 patients with confirmed APDS. Patients taking Joenja saw a reduction in lymph node size and a 37% improvement in naïve B cells counts compared with placebo, indicating a correction of the underlying immune defect.² Joenja has launched with an average wholesale price (AWP) of $54,000 per bottle (60 tablets).
• Mekinist^® (trametinib) + Tafinlar^® (dabrafenib): Novartist/GSK’s Mekinist^® + Tafinlar^® has been approved by the FDA for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. The FDA also approved liquid formulations of Tafinlar and Mekinist. FDA approval was based on the Phase 2/3 TADPOLE trial which demonstrated an ORR of 47% and median progression-free survival (mPFS) of 20.1 months for Tafinlar + Mekinist compared to 11% ORR and 7.4 months mPFS for standard of care.³ Launch and price are pending.
• Omisirge^® (omidubicel-onlv): The FDA has approved Gamida Cell’s allogeneic cell therapy, Omisirge for use in adult and pediatric patients 12 years and older with hematologic malignancies planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection. Omisirge, administered as a single intravenous dose, is composed of human allogeneic stem cells from umbilical cord blood that are processed and cultured with a form of vitamin B3 called nicotinamide. Each dose is patient-specific, containing healthy stem cells from an allogeneic pre-screened donor, meaning it comes from a different individual rather than using the patient’s own cells. Omisirge was approved based on the Phase 3 trial that evaluated the efficacy and safety of Omisirge compared with standard umbilical cord blood transplantation. 87% of patients receiving Omisirge achieved neutrophil recovery with a median of 12 days compared with 83% of those who received umbilical cord blood transplantation and achieved neutrophil recovery with a median of 22 days. Additionally, bacterial or fungal infections by 100 days following transplantation were seen in 39% vs 60% of patients in the Omisirge and control arm, respectively.⁴ Omisirge launch and price are pending.

New Indications

• Hyrimoz^® HCF (adalimumab-adaz): The FDA approved Sandoz’s Hyrimoz HCF (adalimumab-adaz) as a high concentration formulation of Hyrimoz, a Humira biosimilar.
• Keytruda^® (pembrolizumab): The FDA granted a conversion from accelerated to full approval of Merck’s Keytruda for treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and have no satisfactory alternative treatments.
• Livmarli^® (maralixibat): The FDA expanded the indication for Mirum Pharmacueticals’ Livmarli (maralixibat) to include pediatric indication for the minimally absorbed apical sodium bile dependent bile acid transporter (ASBT) to lower the one-year and older age threshold for treatment of cholestatic pruritis in Alagille syndrome (ALGS) to patients aged three months of age and older.
• Evkeeza^® (evinacumab-dgnb): Regeneron’s Evkeeza (evinacumab-dgnb) has been granted approval to expand its indication to be used in adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia.

April News

• “Data Bridge Market Research analyses that the adalimumab biosimilar market which is $598.30 million in 2022, is expected to reach $3,431.48 million by 2030, at a compound annual growth rate of 24.4% during the forecast period 2023 to 2030. In addition to the insights on market scenarios such as market value, growth rate, segmentation, geographical coverage, and major players, the market reports curated by the Data Bridge Market Research also include depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.”⁵
• “Biosimilar entrants are likely to protect Regeneron Pharmaceuticals, Inc.’s Eylea (aflibercept) from price negotiations under the Inflation Reduction Act, though the protection may hinge in part on how successful Regeneron is at switching patients to a planned high-dose formulation of the biologic. If Regeneron successfully moves patients to the new formulation, leaving little market for biosimilars of the older version, Eylea’s exemption from negotiations could pivot on the Centers for Medicare and Medicaid Services definition of “bona fide” marketing, that is whether the U.S. government considers biosimilar competition to the older formulation to represent “bona fide” marketing of Eylea copycats if most patients are no longer taking that formulation.”⁶
• “Gene therapies approved by the FDA have so far brought with them eye-popping prices of $2 million or more because of the extraordinary savings, sometimes over a lifetime, that can occur if the therapies prove safe, effective and durable. In the case of the incoming sickle cell disease gene therapies from Vertex Pharmaceuticals and CRISPR Therapeutics, known as exagamglogene autotemcel or exa-cel, and bluebird bio’s lovotibeglogene autotemcel or lovo-cel, ICER said that for a price of about $2 million per potential shot, the therapies could be cost effective under some scenarios although “these gene therapies have an incremental cost effectiveness that is above commonly cited thresholds from the health care system perspective.””⁷

References

1. https://www.businesswire.com/news/home/20230322005618/en/Incyte-Announces-FDA-Approval-of-Zynyz%E2%84%A2-retifanlimab-dlwr-for-the-Treatment-of-Metastatic-or-Recurrent-Locally-Advanced-Merkel-Cell-Carcinoma-MCC

2. https://www.hcplive.com/view/leniolisib-fda-approved-rare-immunodeficiency-disease
3. https://www.novartis.com/news/media-releases/novartis-tafinlar-mekinist-approved-fda-pediatric-patients-braf-v600e-low-grade-glioma-most-common-pediatric-brain-cancer
4. https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell
5. https://finance.yahoo.com/news/adalimumab-biosimilar-market-probable-influence-180300954.html
6. https://pink.pharmaintelligence.informa.com/PS147990/Will-Biosimilar-Competition-And-Product-Hopping-Save-Regenerons-Eylea-Or-Cancel-Each-Other-Out
7. https://endpts.com/at-2m-per-shot-icer-says-two-new-sickle-cell-gene-therapies-could-be-cost-effective/