More than half of dermatomyositis patients are not optimally managed, according to a survey of 102 U.S. rheumatologists and dermatologists by Spherix Global Insights.
Currently, the standard of care predominantly involves corticosteroids, topical therapies, traditional disease-modifying anti-rheumatic drugs (tDMARDs), and, in more severe or refractory cases, intravenous immunoglobulin (IVIg) or off-label biologics, underscoring the growing unmet need for novel therapeutic options to treat this condition.
Specialists confirm that corticosteroids remain the most frequently prescribed first-line treatment for DM, followed by traditional disease-modifying anti-rheumatic drugs such as methotrexate and mycophenolate mofetil (tDMARDs), as well as intravenous immunoglobulin (IVIg) and off-label biologics like rituximab.
However, treatment approaches are evolving, with over two-thirds of rheumatologists and nearly half of dermatologists reporting changes in their management of DM over the past year. These shifts often involve increased or earlier use of IVIg, more frequent patient visits, and a growing reliance on off-label advanced systemic therapies aimed at reducing steroid dependence.
In the absence of therapeutic advancements, the vast majority of rheumatologists and dermatologists point to an extremely high unmet need for new pharmacological treatment options for DM. As one respondent highlighted, “[the greatest challenge in managing DM is the] lack of safe and tolerable medications – many have seriously bad safety profiles and poor risk-reward ratios.”
Survey responders emphasize there is a pressing need for a safe, effective agent that is steroid-sparing, addresses both skin and muscle involvement, and that targets the underlying causes of the disease rather than just alleviating symptoms. Furthermore, respondents report that the majority of their classic DM and half of clinically amyopathic dermatomyositis (CADM) patients would be considered candidates for new advanced systemic therapies.
What’s in the DM pipeline?
Focusing on the pipeline, respondents were provided with target product profiles for anifrolumab-fnia (Saphnelo, AstraZeneca) Priovant Therapeutics’ JAK1/TYK2 brepocitinib, Pfizer’s dazukibart, CSL Behring’s Hizentra (Immune Globulin Subcutaneous [Human] 20% Liquid) and Amgen/Horizon’s daxdilimab to gauge specialist feedback on overall likelihood to prescribe, degree of advancement, patient candidacy, and anticipated timeline for use upon approval.
Rheumatologists favored anifrolumab-fnia due to their familiarity with the agent in systemic lupus erythematosus (SLE), and dermatologists leaning towards Priovant’s brepocitinib, valuing its oral route of administration and unique but familiar mechanism of action.
While Saphnelo and brepocitinib have garnered higher rates of preference, CSL Behring’s Hizentra may also play a pivotal role in filling the noted unmet need, if approved. Unlike other immune globulin agents currently in use like Pfizer’s Octagam 10%, Hizentra offers the convenience of subcutaneous injection, eliminating the need for IV access—a key advantage for DM treaters, with over half expressing a preference for new therapies that can be administered via injection at home.
Specialists viewed the convenience of Hizentra’s subcutaneous dosing as a significant benefit, along with its mechanism of action and its proven efficacy. Consequently, it’s not surprising that a majority anticipate trialing Hizentra within the first six months of its potential approval, estimating a sizeable portion of their DM patients would be suitable candidates. As one specialist reports, “[It is a] familiar MOA, I would feel comfortable prescribing and feel it is definitely indicated/justifiable depending on the extent of disease.”
Source: The Dermatology Digest- thedermdigest.com/survey-u-s-derms-and-rheums-report-extremely-high-unmet-need-for-new-treatments-for-dm/
