Reports & eBooks

Drug Pipeline & Clinical Update - January 2025

February 10, 2025

Capital Rx

Highlights

  • Four key new drug approvals, including Tryngolza™ oral solution to adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)
  • Eight notable expanded indications, including Zepbound™ (tirzepatide) injection – expanded indication, in combination with a reduced-calorie diet and increased physical activity, for the treatment of moderate-to-severe obstructive sleep apnea in adults with obesity
  • One biosimilar approval: Steqeyma™ (ustekinumab-stba) subcutaneous injection for the treatment of Crohn’s disease, ulcerative colitis, plaque psoriasis, psoriatic arthritis
  • Capital Rx's Updated Drug Recall Report: CLICK HERE to review

Capital Rx's Clinical Team closely monitors the drug landscape to provide our clients with timely information on newly FDA-approved medications, as well as products in the pipeline. Read on to review our January 2025 Monthly Drug Update!

Key New Drug Approvals

Ensacove™ (ensartinib) capsule

Approval Date: 12/18/2024

Indication: Treatment of adult patients with anaplastic lymphoma kinase (ALK)-positivelocally advanced or metastatic non-small cell lung cancer (NSCLC) who have not previously received an ALK inhibitor

Cost*: TBD

Key Consideration:
• Sixth ALK inhibitor FDA-approved for ALK-positive NSCLC, with Alecensa leading the market share in the class
• Recommend dosage is 225 mg orally once daily with or without food until disease progression or unacceptable toxicity

Press Release: https://www.businesswire.com/news/home/20241218355621/en/FDA-Approval-of-Ensartinib-for-ALK-Positive-Locally-Advanced-or-Metastatic-Non-Small-Cell-Lung-Cancer-NSCLC

Tryngolza™ (olezarsen) injection

Approval Date: 12/19/2024

Indication: Adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)

Cost*: $595,000annually

Key Consideration:
• FCS is a rare genetic form of severe hypertriglyceridemia and can lead to acute pancreatitis if not treated or undertreated
• First FDA-approved treatment for FCS, however competition is anticipated, as the FDA is currently reviewing a new drug application for plozasiran, with possible approval in Q3 2025
• Available as an 80 mg single-dose autoinjector; administered subcutaneously once monthly

Press Release:  https://ir.ionis.com/news-releases/news-release-details/tryngolzatm-olezarsen-approved-us-first-ever-treatment-adults

Alyftrek™ (deutivacaftor/tezacaftor/vanzacaftor) tablet

Approval Date: 12/20/2024

Indication: Treatment of cystic fibrosis in patients aged 6 years and older who have at least one F508delmutation or another responsive mutation in the CFTR gene

Cost*: $370,270 annually

Key Consideration:
• Fifth FDA-approved CFTR-modulating agent manufactured by Vertex, who also manufactures Trikafta™, Symdeko™, Orkambi™, and Kalydeco™
• Alyftrek represents a notable clinical improvement over Trikafta with once-daily dosing, as well as improvements in reducing sweat chloride levels

Press Release:  https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-alyftrektm-once-daily-next

Alhemo™ (concizumab-mtci) injection

Approval Date: 12/20/2024

Indication: Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adultand pediatric patients 12 years of age and older with hemophilia A with FVIII inhibitors or hemophilia B with FIX inhibitors

Cost*: TBD

Key Consideration:
• Second tissue factor pathway inhibitor (TFPI) antagonist approved for the treatment of hemophilia A and B; Hympavzi™, a once-weekly subcutaneous TFPI antagonist, was approved in October 2024 for the same indication
• Administered subcutaneously once daily via a single-patient-use prefilled pen that is available in three strengths; dosing is determined by weight and response to initial treatment

Press Release: https://www.novonordisk-us.com/media/news-archive/news-details.html?id=915084

Notable Expanded Indications

• Gemtesa™ (vibegron) tablet – expanded indication for the treatment of overactive bladder with symptoms of urge urinary incontinence, urgency, and urinary frequency in adult males on pharmacological therapy for benign prostatic hyperplasia (BPH)

• Trikafta™ (elexacaftor/tezacaftor/ivacaftor and ivacaftor) tablet and oral granule – expanded indication to include an additional 94 non-F508del CFTR mutations for the treatment of people with cystic fibrosis ages 2 and older who have at least one F508del mutation in the CFTR gene or a mutation that is responsive to Trikafta based on clinical and/or in vitro data

• Braftovi™ (encorafenib) capsule – expanded indication, in combination with cetuximab and mFOLFOX6, for the treatment of patients with metastatic colorectal cancer with a BRAF V600E mutation, as detected by an FDA-approved test

• Zepbound™ (tirzepatide) injection – expanded indication, in combination with a reduced-calorie diet and increased physical activity, for the treatment of moderate-to-severe obstructive sleep apnea in adults with obesity

• Omvoh™ (mirikizumab-mrkz) injection – expanded indication for the treatment of moderately to severely active Crohn’s disease in adults

• Calquence™ (acalabrutinib) capsule and tablet – expanded indication, in combination with bendamustine and rituximab, for the treatment of adult patients with previously untreated mantle cell lymphoma who are ineligible for autologous hematopoietic stem cell transplantation

• Lumakras™ (sotorasib) tablet – expanded indication, in combination with Vectibix™, for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer who have received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy

• Spravato™ (esketamine) nasal spray – expanded indication for the treatment of treatment-resistant depression in adults, as monotherapy or in conjunction with an oral antidepressant

Biosimilar Approvals

Steqeyma™ (ustekinumab-stba) subcutaneous injection

Approval Date: 12/17/2024

Indication: Treatment of Crohn’s disease, ulcerative colitis, plaque psoriasis, psoriatic arthritis

Additional Information:
• Biosimilar to Stelara™ (ustekinumab)
• Intravenous (IV) formulation also FDA approved for the same indications
• Seventh Stelara™ biosimilar approved; anticipated launch in March 2025

Press Release: https://www.prnewswire.com/news-releases/us-fda-approves-celltrions-steqeyma-ustekinumab-stba-a-biosimilar-to-stelara-ustekinumab-302334458.html

Generic Launches

• Betimol™ (timolol 0.5%) ophthalmic solution
Motegrity™ (prucalopride 1 mg, 2 mg) tablet
• Victoza™ (liraglutide 18 mg/3 mL) injection
• Nexium™ (esomeprazole 2.5 mg, 5 mg) packet
• Namzaric™ (memantine-donepezil 28-10 mg, 14-10 mg) extended-release capsule

FDA Safety Alert

• The FDA has required and approved safety labeling changes to the prescribing information for Abrysvo™ and Arexvy™, both respiratory syncytial virus (RSV) vaccines, to include the following language in the Warnings and Precautions section:
     o “The results of a postmarketing observational study suggest an increased risk of Guillain-Barré syndrome during the 42 days following vaccination”

Contact Us if you'd like to learn more about Capital Rx's full-service pharmacy benefit management (PBM) solutions and clinical programs.

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* Cost: actual patient out-of-pocket costs may be lower, as the list price does not reflect insurance coverage, co-pay support for eligible patients, or financial assistance from patient support

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