Specialty Drug Pipeline Quarterly Update: June 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 articles for pipeline information on traditional drugs, biosimilars and gene/cell therapy.

New Drug Information

• Columvi^® (glofitamab-gxbm): The U.S. Food and Drug Administration (FDA) has granted accelerated approval Genentech’s Columvi for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified or large B-cell lymphoma (LBCL) arising from follicular lymphoma, after two or more lines of systemic therapy. Columvi was approved based on an overall response rate of 56% and 43% of patients achieving a complete response durability of response in the Phase 1/2 NP30179 study. Sixty-eight percent of patients who responded continued to respond for at least nine months. The median duration of response was 1.5 years.¹ Columvi launch and price are pending.
• Vyvgart^® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc): The FDA has approved Halozyme Therapeutics’ Vyvgart Hytrulo injection for subcutaneous (SC) use for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. Vyvgart^® Hytrulo is a subcutaneous product combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as Vyvgart^®, and recombinant human hyaluronidase PH20 (rHuPH20). The product is to be administered subcutaneously by a healthcare professional as a single injection (1,008 mg fixed dose) over 30-90 seconds in cycles of once weekly injections for four weeks.  Vyvgart Hytrulo was approved based on the Phase 3 ADAPT-SC study that compared the efficacy and safety of SC efgartigimod to IV efgartigimod in adults with gMG. Results demonstrated that treatment with Vyvgart Hytrulo met the primary endpoint of noninferiority to IV efgartigimod at day 29. Patients treated with Vyvgart Hytrulo achieved a mean total IgG reduction of 66.4% compared with a 62.2% reduction for IV efgartigimod.² Launch and price are pending.

New Indications

• Bylvay^® (odevibat): The FDA has expanded Albireo’s Bylvay to include a new indication for the non-systemic ileal bile acid transport inhibitor (IBATi) for treatment of cholestatic pruritis in patients aged 12 months and older with Alagille syndrome (ALGS).
• Lynparza^® (olaparib): The FDA approved AstraZeneca’s Lynparza (olaparib) to be used in combination with abiraterone and prednisone or prednisolone for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC).
• Rinvoq^® (upadacitinib): AbbVie’s Rinvoq (upadacitinib) was approved by the FDA to include for treatment of adults with moderately to severely active Crohn’s disease.

June News

• “The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6-0 in favor of fully approving Eisai and Biogen’s Alzheimer’s drug Leqembi (lecanemab), which first won an accelerated approval in January. The full approval, expected on or before July 6, would open the door to wider insurance and Medicare coverage for those with mild cognitive impairment or mild dementia and confirmed presence of amyloid beta pathology but who could not or do not want to pay the $26,500 per year list price.”3
• “The FDA has granted priority and standard review designations to the biologics license applications for exagamglogene autotemcel (exa-cel; formerly CTX001) for the treatment of patients with sickle cell disease and transfusion-dependent beta thalassemia, respectively, marking the first CRISPR gene editing filings to be accepted for review by the FDA. The FDA has set action dates of December 8, 2023, for the sickle cell indication, and March 30, 2024, for the transfusion-dependent beta thalassemia indication.”4
• “Merck is teasing data for efinopegdutide, a GLP-1/glucagon receptor co-agonist, in nonalcoholic steatohepatitis (NASH) and how it compares to Novo Nordisk’s semaglutide, with plans to share more detailed data from the study at the European Association for the Study of the Liver’s annual meeting later this month. Merck plans to use data from efinopegdutide’s Phase IIa trial in adult patients with nonalcoholic fatty liver disease to inform a Phase IIb study set to begin later this month in NASH, a more severe form of the disease, according to Merck. The FDA recently granted the drug, which is also being evaluated as a treatment for obesity and diabetes, a fast track designation in NASH.”⁴

References

1. https://www.gene.com/media/news-features/fda-approves-genentechs-fixed-duration-bispecific-antibody-for-relapsed-refractory-diffuse-large-b-cell-lymphoma#:~:text=On%20June%2015%2C%202023%2C%20the,more%20lines%20of%20systemic%20therapy.
2. https://www.prnewswire.com/news-releases/halozyme-announces-argenx-receives-fda-approval-for-vyvgart-hytrulo-with-enhanze-for-subcutaneous-use-in-generalized-myasthenia-gravis-301855994.html
3. https://endpts.com/fda-adcomm-votes-unanimously-in-favor-of-full-approval-for-eisais-new-alzheimers-drug/
4. https://www.onclive.com/view/fda-set-to-review-exagamglogene-autotemcel-for-sickle-cell-disease-transfusion-dependent-beta-thalassemia
5. https://endpts.com/merck-steps-into-liver-disease-space-with-early-data-for-its-glp-1-glucagon-receptor-co-agonist/