Specialty Drug Pipeline Quarterly Update: September 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

• Aphexda^™ (motixafortide): BioLine Therapeutics’ Aphexda was FDA approved in combination with filgrastim, a granulocyte stimulating factor) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation (ASCT) in patients with multiple myeloma (MM). Aphexda is administered subcutaneously by a health care professional 10 to 14 hours prior to beginning apheresis. In the Phase 3 GENESIS trial, 67.5% of patients treated with Aphexda achieved the stem cell collection goal within two apheresis sessions versus 9.5% of patients who received filgrastim alone, as measured by central laboratory. Mozobil^® (plerixafor) and its generic versions which were launched in July 2023, share a similar indication. Launch of Aphexda is planned in September 2023 with pricing to follow.¹
• Ojjaara™ (momelotinib): GlaxoSmithKline’s oral, once-daily Ojjaara received FDA approval for the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF [postpolycythemia vera (PV) and post-essential thrombocythemia (ET)], in adults with anemia.[i] Ojjaara is a first-in-class inhibitor of Janus kinase 1 and 2 (JAK1/2) and activin A receptor type 1 (ACVR1) and first JAK inhibitor to treat myelofibrosis in patients with anemia. In the phase 3 MOMENTUM trial, momelotinib was superior to danazol in the primary endpoint of the proportion of patients achieving a ≥ 50% reduction from baseline in the Myelofibrosis Symptom Assessment Form Total Symptom Score (MSA TSS; 25% versus 9%, respectively; p=0.0095).² The wholesale acquisition cost (WAC) for Ojjaara will be $322,800. Ojjaara will compete for market share with: Jakafi (ruxolitinib), Inrebic (fedrantinib), and Vonjo (pancritinib). Ojjaara is the only one approved for both newly diagnosed and previously treated myelofibrosis patients with anemia; however, in a Phase 3 trial, SIMPLIFY-1, Ojjaara was non-inferior to Jakafi in the primary endpoint (splenic volume reduction) but failed to improve disease-associated symptoms compared with Jakafi.³

 New Indications

• Reblozyl^® (luspatercept-aamt): The FDA approved a new indication for Bristol-Myers Squibb’s Reblozyl for the treatment of anemia without previous use of an erythropoiesis stimulating agent use (ESA-naïve) in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions.
• Mekinist^® (trametinib) plus Tafinlar^® (dabrafenib): The FDA expanded the indications for Novartis’ Mekinist and Tafinlar for their combined use to treat patients with unresectable or metastatic solid tumors with BRAF V600E mutation that has progressed following prior treatment and with no satisfactory alternative treatment options to include patients 1 to 5 years of age. The indications were approved under Accelerated Approval based on overall response rate and duration of response; continued approval may depend upon results from confirmatory trial(s).
• Temodar^® (temozolomide): The FDA approved Merck’s Temodar for a new indication for adjuvant treatment of adults with newly diagnosed anaplastic astrocytoma. The FDA also revised the existing refractory anaplastic astrocytoma indication to remove the requirement that patients had experienced disease progression while on a procarbazine- and nitrosourea-containing drug regimen.

References

1. BioLineRx’s Aphexda receives FDA approval for stem cell mobilisation (msn.com)
2. https://www.formularywatch.com/view/fda-approves-gsk-s-myelofibrosis-drug
3. https://ashpublications.org/ashclinicalnews/news/3197/Mixed-Results-in-Trials-of-Momelotinib-Versus