Drug Pipeline & Clinical Update - September 2025

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 September 2025 Monthly Drug Update!

Key New Drug Approvals

Dawnzera™ (donidalorsen) subcutaneous injection

Approval Date: 8/21/2025

Indication: Prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older

Cost*: ~$750,000 annually

Key Consideration:
• The first and only RNA-targeted therapy to prevent HAE attacks
• Dosed every 4 or 8 weeks, offering the longest dosing option for HAE
• Received Orphan Drug Designation by the FDA

‍Press Release: https://ir.ionis.com/news-releases/news-release-details/dawnzeratm-donidalorsen-approved-us-first-and-only-rna-targeted

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Wayrilz™ (rilzabrutinib) oral tablet

Approval Date: 8/29/2025

Indication: Treatment of persistent or chronic immune thrombocytopenia (ITP) in adults who have had an insufficient response to a previous treatment

Cost*: $17,500 per month

Key Consideration:
• First BTK inhibitor to be approved for ITP
• Received Orphan Drug Designation by the FDA

‍Press Release: https://www.sanofi.com/en/media-room/press-releases/2025/2025-08-29-21-50-18-3141825

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Leqembi® Iqlik™ (lecanemab-irmb) subcutaneous injection

Approval Date: 8/29/2025

Indication: Treatment of Alzheimer’s disease

Cost*: $19,500 annually

Key Consideration:
• The first and only anti-amyloid subcutaneous injection approved for Alzheimer’s disease
• Requires 18 months of IV Leqembi® use prior to switching to Leqembi® Iqlik^TM

‍Press Release: https://investors.biogen.com/news-releases/news-release-details/fda-approves-leqembir-iqliktm-lecanemab-irmb-subcutaneous

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Embumyst™ (bumetanide) nasal spray

Approval Date: 9/12/2025

Indication: Treatment of edema associated with congestive heart failure, renal and hepatic disease in adults

Cost*: TBD

Key Consideration:
• First and only nasal spray formulation of a loop diuretic approved by the FDA
• Not intended for chronic use; intended to help bridge the gap between oral and IV diuretics

‍Press Release: https://www.businesswire.com/news/home/20250915243998/en/Corstasis-Therapeutics-Announces-FDA-Approval-of-ENBUMYST-bumetanide-nasal-spray-for-the-Treatment-of-Edema-Associated-with-Congestive-Heart-Failure-Liver-Disease-and-Kidney-Disease

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Subvenite® (lamotrigine) oral suspension

Approval Date: 9/16/2025

Indication: Treatment of epilepsy in patients 2 years of age and older; treatment of bipolar disorder

Cost*: TBD

Key Consideration:
• The first and only lamotrigine suspension approved by the FDA
• Stored at room temperature; stable for 90 days after first opening

‍Press Release: https://firstwordpharma.com/story/6093514

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Forzinity™ (elamipretide) subcutaneous injection

Approval Date: 9/19/2025

Indication: Improve muscle strength in patients with Barth syndrome weighing at least 30 kg

Cost*: TBD

Key Consideration:
• The first and only treatment approved for Barth syndrome
• The first approved mitochondrial-targeted therapy
• Received Priority Review and Orphan Drug Designation by the FDA

‍Press Release: https://stealthbt.com/stealth-biotherapeutics-announces-fda-accelerated-approval-of-forzinity-elamipretide-hcl-the-first-therapy-for-progressive-and-life-limiting-ultra-rare-genetic-disease-barth-syndrome/

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Notable Expanded Indications

• Koselugo® (selumetinib) – expanded to treat neurofibromatosis type 1 (NF1) in patients 1 year of age and older who have symptomatic, inoperable plexiform neurofibromas (PN)
• Opzelura® (ruxolitinib) – expanded to treat mild to moderate atopic dermatitis in non-immunocompromised patients 2 years of age and older

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‍‍Generic Launches

• Saxenda® (liraglutide) subcutaneous injection

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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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