Specialty Pipeline Update: August 2022

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

• Cimerli^® (ranibizumab-eqrn): The U.S. Food and Drug Administration (FDA) has approved Cimerli as a biosimilar product interchangeable with Lucentis^® (ranibizumab injection) for all five indications, include neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy (DR), and myopic choroidal neovascularization (mCNV). Cimerli met the FDA’s rigorous standards to the reference product, including safety, efficacy and quality.¹Cimerli belongs to the anti-VEGF therapy class of biologics that has been revolutionary in helping retinal patients maintain or gain vision. Cimerli will launch in both 0.3mg and 0.5mg dosages, in early October 2022 with pricing to follow.
• Zynteglo^®(betibeglogene autotemcel): The FDA has approved Bluebird bio’s Zynteglo also known as beti-cel as a one-time gene therapy to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. Beta-thalassemia, a type of inherited blood disorder, reduces the levels of normal hemoglobin and red blood cells via mutations in the beta-globin subunit; this can ultimately cause the delivery of oxygen throughout the body to be at insufficient levels. The subsequently low levels of red blood cells can result in a number of health problems, such as dizziness, weakness, fatigue, bone abnormalities, and other more serious complications. Zynteglo established the efficacy based on the achievement of transfusion independence, which is the point at which the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Across two clinical studies evaluating adults and kids with transfusion-dependent beta thalassemia, 89% of the 41 patients on Zynteglo achieved transfusion independence.² Bluebird bio anticipates its launch in 2023 with a one-time price of $2.8 million per patient.³ The list price doesn’t include the cost of hospitalization required for patients receiving the therapy.

New Indications

• Krystexxa^® (pegloticase) + methotrexate: The FDA expanded Horizon’s Krystexxa (pegloticase) + methotrexate indication to include a co-administration with methotrexate for the treatment of chronic gout.
• Opzelura^® (ruxolitinib topical cream): Incyte’s Opzelura has been granted a new indication by the FDA to include treatment of nonsegmental vitiligo in adults and adolescents 12 year of age and older.
• Benlysta^® (belimumab): The FDA granted GlaxoSmithKline’s Benlysta to include a new pediatric indication for the B-lymphocyte stimulator (BLyS)-specific inhibitor for IV treatment of children aged five to 17 years old with active lupus nephritis who are receiving standard therapy.
• Stelara^® (ustekinumab): Johnson & Johnson (Janssen)’s Stelara has an expanded indication granted by the FDA for the antibody targeting interleukin-12 and IL-23 for treatment of pediatric patients aged six years and older with psoriatic arthritis (PsA).
• Enhertu^® (fam-trastuzumab deruxtecan-nxki): The FDA has expanded the indication of Daiichi Sankyo and AstraZeneca’s Enhertu to include the treatment of patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH) breast cancer who have received a prior chemotherapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy. Additionally, Enhertu was approved to treat HER2-positive non-small cell lung cancer who have received a prior systemic therapy.
• Nubeqa^® (darolutamide): Bayer and Orion’s Nubeqa was granted a new indication by the FDA for use in combination with docetaxel for treatment of metastatic hormone-sensitive prostate cancer (mHSPC).
• Tabrecta^® (capmatinib): The FDA expanded the indication of Novartis’ Tabrecta to include the treatment of adults with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test.

August News

• “Eli Lilly and Co said it plans to make its COVID-19 antibody drug commercially available to U.S. states as well as hospitals and other healthcare providers from August. The drug, bebtelovimab, had received authorization in the United States in February for the treatment of mild-to-moderate COVID-19 among adults and children. Eli Lilly entered an agreement in June to supply an additional 150,000 doses of the drug to the U.S. government.”⁴
• “The FDA has accepted a biologics license application for and granted priority review to omidubicel as a potential treatment for patients with blood cancers who need an allogeneic hematopoietic stem cell transplant, according to a press release from Gamida Cell. The application is supported by results from a Phase 3 trial (NCT02730299) that assessed the safety and efficacy of omidubicel vs standard umbilical cord blood transplant in patients with hematologic malignancies such as lymphoma, leukemia, and myelodysplastic syndrome. The FDA has set a prescription drug user fee act date of January 30, 2023.”⁵
• “Another summer day, another Enhertu update. The AstraZeneca-Daiichi Sankyo antibody-drug conjugate has passed another trial, this time a Phase 3 trial testing the cancer drug versus physician’s choice in patients with HER2-positive metastatic breast cancer. The DESTINY-Breast02 study beat out the other treatments — trastuzumab/capecitabine or lapatinib/capecitabine — in improving progression-free survival and a secondary endpoint, overall survival.”⁶
• “Novartis has recorded two deaths after treatment with its spinal muscular atrophy gene therapy Zolgensma, once again bringing gene therapy’s safety into attention. Two children in Russia and Kazakhstan died about five to six weeks after receiving Zolgensma, Novartis confirmed to Fierce Pharma. Both patients died of acute liver failure, a known side effect of Zolgensma that’s included in a boxed warning on the one-time therapy’s label. Both had received corticosteroid taper to restore liver function.”⁷

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References

1. https://www.globenewswire.com/news-release/2022/08/02/2490955/33333/en/FDA-Approves-Coherus-CIMERLI-ranibizumab-eqrn-as-the-First-and-Only-Interchangeable-Biosimilar-to-Lucentis-for-All-Five-Indications-with-12-Months-of-Interchangeability-Exclusivity.html
2. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-treat-adult-and-pediatric-patients-beta-thalassemia-who
3. https://www.fiercepharma.com/pharma/bluebirds-28m-gene-therapy-zynteglo-gets-fda-backing-beta-thalassemia
4. https://www.reuters.com/business/healthcare-pharmaceuticals/eli-lilly-make-covid-19-antibody-drug-commercially-available-august-2022-08-03/
5. https://www.cancernetwork.com/view/fda-accepts-bla-and-grants-priority-review-to-omidubicel-in-hematologic-malignancies
6. https://endpts.com/enhertu-strikes-again-with-a-phiii-success-in-another-breast-cancer-population/
7. https://www.fiercepharma.com/pharma/two-deaths-after-novartis-zolgensma-bring-gene-therapys-liver-safety-spotlight-again?utm_source=email&utm_medium=email&utm_campaign=LS-NL-FiercePharma&oly_enc_id=6999D8336467C2P