May 2024 decisions expected from the FDA

Your monthly synopsis of new drugs expected to hit the market

April 15, 2024

Your monthly synopsis of new drugs expected to hit the market

FDA DECISIONS EXPECTED

At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the United States (U.S.) Food and Drug Administration (FDA).

Drug pipeline for May 2024:

May 2024 rituximab biosimilar (DRL_RI)

Dr. Reddy’s Laboratory is seeking FDA approval for their rituximab biosimilar candidate to Genentech’s Rituxan®, a cluster of differentiation 20 (CD20)-directed cytolytic antibody indicated for the treatment of rheumatoid arthritis, non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, pemphigus vulgaris, granulomatosis with polyangiitis and microscopic polyangiitis. If approved, DRL_RI will be the fourth biosimilar approved in the U.S. for Rituxan.

5/10/2024 Respiratory syncytial virus (RSV) prefusion F protein vaccine (mRNA-1345)

Using a Priority Review voucher, Moderna submitted their mRNA-1345 vaccine for the prevention of RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD) in adults aged 60 years or older. The phase 2/3 ConquerRSV trial demonstrated a vaccine efficacy (VE) of 83.7% (p<0.0001) against RSV-LRTD with ≥ 2 symptoms and a VE of 82.4% (p=0.0078) against RSV-LRTD with ≥ 3 symptoms (co-primary endpoints) at a median follow-up of 112 days after a single IM dose.¹ The vaccine received Breakthrough Therapy and Fast Track status and a Priority Review from the FDA. If approved, mRNA-1345 will be the first RSV vaccine that uses mRNA technology and will compete with Arexvy™ and Abrysvo™ for the prevention of RSV-LRTD in older adults.

For more information, see the mRNA RSV vaccine Deep Dive in the January 2024 edition of the Quarterly Pipeline. Quarterly Drug Pipeline – Prime Therapeutics LLC

5/14/2024 palopegteriparatide (TransCon PTH)

The FDA is reviewing the new drug application (NDA) for palopegteriparatide, a prodrug of parathyroid hormone (PTH [1-34]), for the treatment of hypoparathyroidism in adults. Ascendis resubmitted the NDA after receiving a Complete Response Letter (CRL) from the FDA in May 2023. Notably, once daily administration of palopegteriparatide provides PTH levels in the physiological range over 24 hours. The phase 3 PaTHway trial reported 81% of patients treated with palopegteriparatide achieved both normal serum calcium and independence from conventional therapy at 52 weeks.² Palopegteriparatide has Orphan Drug status.

5/16/2024 rivoceranib + camrelizumab

Elvar is awaiting FDA approval of their oral tyrosine kinase inhibitor, rivoceranib, for use in combination with Jiangsu Hengrui’s investigational IV programed cell death-1 inhibitor, camrelizumab, as a first-line treatment of unresectable hepatocellular carcinoma (uHCC). In the open-label, phase 3 CARES 310 trial, rivoceranib + camrelizumab provided statistically significant and clinically meaningful prolonged overall survival and progression-free survival, compared oral sorafenib.³ If approved, rivoceranib + camrelizumab may compete with the standard of care for uHCC (atezolizumab + bevacizumab). Rivoceranib and camrelizumab were granted Orphan Drug designation by the FDA for uHCC.

For more information, see the rivoceranib_camrelizumab Deep Dive in the January 2024 edition of the Quarterly Pipeline. Quarterly Drug Pipeline – Prime Therapeutics LLC

5/25/2024 prademagene zamikeracel (pz-cel)

The dermatologic gene therapy by Abeona, prademagene zamikeracel, has been submitted to the FDA for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The FDA granted the product Breakthrough Therapy, Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy designations, as well as a Priority Review. The ex vivo cell therapy is created from skin biopsies that are engineered into autologous keratinocyte sheets that contain the COL7A1 gene. The sheets are engrafted as a one-time surgical procedure under anesthesia in a hospital setting. Top-line data from the open-label, phase 3 VIITAL trial revealed, at six months, significantly more wounds achieved at least 50% healing from baseline when treated with pz-cel compared to untreated control wounds (81.4% versus16.3%, respectively; p<0.0001) and wound dressing changes were significantly less painful for patients who received pz-cel.4 If approved, pz-cel will be the second gene therapy, and third treatment overall, approved for epidermolysis bullosa. It will compete with the topical gene therapy Vyjuvek® in patients with RDEB.

For more information, see the prademagene zamikeracel Deep Dive in the January 2024 edition of the Quarterly Pipeline. Quarterly Drug Pipeline – Prime Therapeutics LLC

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