Reports & eBooks

Drug Pipeline & Clinical Update - December 2024

January 6, 2025

Capital Rx

Highlights

  • Three key new drug approvals, including Imkeldi™(imatinib) oral solution for the treatment of certain forms of leukemia and other cancers
  • Three notable expanded indications, including Vtama™ (tapinarof) cream, for treatment of atopic dermatitis in adults and pediatric patients >2 years of age
  • One biosimilar approval: Yesintek™ (ustekinumab-kfce) subcutaneous injection
  • 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 December 2024 Monthly Drug Update!

Key New Drug Approvals

Attruby™ (acoramidis) tablet

Approval Date: 11/22/2024

Indication: Treatment of cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization

Cost*: $244,500 annually

Key Consideration:
• Received orphan drug designation
• Recommended dosage is 712 mg orally twice daily; available as a therapy pack containing 4 28-blister cards (112 tablets)
• Expected to compete with Vyndaqel™ (tafamidis meglumine) and VyndamaxTM (tafamidis); both were FDA approved in 2019 for ATTR-CM

Press Release: https://investor.bridgebio.com/news-releases/news-release-details/attrubytm-acoramidis-near-complete-ttr-stabilizer-90-approved

Imkeldi™ (imatinib) oral solution

Approval Date: 11/22/2024

Indication: Treatment of certain forms of leukemia and other cancers

Cost*: TBD

Key Consideration:
• First oral liquid form of imatinib
• Available as a strawberry flavored 80 mg/mL concentrated oral solution; designed to provide dosing accuracy

Press Release: https://shorlaoncology.com/shorla-oncology-announces-fda-approval-of-imkeldi-imatinib-mesylate/

Crenessity™ (crinecerfont) capsule and oral solution

Approval Date: 12/13/2024

Indication: Adjunctive treatment to glucocorticoid replacement to control androgens in adults and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH)

Cost*: TBD

Key Consideration:
• Received priority review and orphan drug designation
• First treatment for classic CAH to directly reduce excess adrenocorticotropic hormone and downstream adrenal androgen production, allowing for glucocorticoid dose reduction
• Recommended dosage is 100 mg orally twice daily for adults; dosage is weight-based for pediatric patients

Press Release: https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-fda-approval-of-crenessity-crinecerfont-a-first-in-class-treatment-for-children-and-adults-with-classic-congenital-adrenal-hyperplasia-302331772.html

Revuforj™ (revumenib) tablet

Approval Date: 11/15/2024

Indication: Treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older

Cost*: $39,500 per month

Key Consideration:
• FDA granted Breakthrough Therapy, Fast Track Designation, and Priority Review
• First and only menin inhibitor; dosed orally twice daily on an empty stomach or with a low-fat meal
• KMT2A generic abnormality is found in about 10% of acute leukemias

Press Release:  https://ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-first-and-only

Notable Expanded Indications

Bimzelx™ (bimekizumab-bkzx) injection – expanded indication to the treatment of adults with moderate-to-severe hidradenitis suppurativa

• Vtama™ (tapinarof) cream – expanded indication to the treatment of atopic dermatitis in adults and pediatric patients 2 years of age and older

• Nemluvio™ (nemolizumab) injection – expanded indication to the treatment of patients 12 years and older with moderate-to-severe atopic dermatitis, in combination with topical corticosteroids and/or calcineurin inhibitors when the disease is not adequately controlled with topical prescription therapies

Biosimilar Approvals

Yesintek™ (ustekinumab-kfce) subcutaneous injection

Approval Date: 12/1/2024

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

Cost*: TBD

Key Consideration:
• Biosimilar to Stelara™ (ustekinumab)
• Intravenous (IV) formulation also FDA approved for the same indications
• Sixth Stelara™ biosimilar approved; anticipated launch in February 2025

Press Release: https://www.biocon.com/u-s-fda-approves-biocon-biologics-yesintek-bmab-1200-biosimilar-to-jjs-stelara-ustekinumab/

Generic Launches

• Betimol™ (timolol 0.5%) ophthalmic solution

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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