骨髄線維症市場：KOLの洞察

当レポートは、世界有数のKOLの専門知識に基づき、骨髄線維症治療の展望に関する公平でバランスの取れた洞察を提供し、市場ダイナミクスを形成する重要な争点に焦点を当てています。KOLが上市された治療薬やパイプライン治療薬の可能性を批判的に評価し、製品のポジショニングと競合に関する戦略的意思決定の指針となる実用的な情報を提供することで、現在および将来の治療パラダイムをより深く理解することができます。

レポートの内容

• 2023年、FDAは血小板数50 x 109/L未満の骨髄線維症（MF）患者に対するエンペキシク（パクリチニブ；CTI Biopharma）の使用を希少疾病用医薬品（オーファンドラッグ）に認定したが治療にどのような影響を与えているか
• Jakafi（ルキソリチニブ；Incyte Coorporation/Novartis）の使用は、過去12カ月間でどのように変化したか
• 第III相FREEDOM2試験が2024年10月に発表され、MF患者（ルキソリチニブ別前治療歴あり）において、入手可能な最善の治療法に対して有効性が向上したと報告されたが、この試験はフェドランチニブの使用にどのような影響を与えるか
• 貧血患者に対するルキソリチニブまたは他の治療法に取って代わるオジャアラの可能性はどの程度あるか
• 骨髄線維症の治療におけるテロメラーゼ阻害剤の可能性と、JAK阻害剤治療が奏効しない患者にとって、テロメラーゼ阻害剤は良い選択肢になるか
• 骨髄線維症の治療において、アンメットニーズは何か、人工知能（AI）ツールやデジタル技術は、これらの懸念に対応できるか
• 第Ⅱ相試験であるSENTRY-2では、JAK阻害剤ナイーブの骨髄線維症患者を対象に、Xpovioの有効性と安全性を評価し、脾臓縮小を主要アウトカムとして承認済みのJAK阻害剤と比較しているが、この試験にはどのような期待があるか
• Syntaraは、第Ⅱ相SNT-5505試験の良好なデータを発表し、12週の時点で、患者の46％が全身スコアが50％減少し、38週の時点では80％に増加。このデータは説得力があるが、他の薬剤と比較してどうか
• KER050はルスパテルセプトのような類似薬と比較してどうか
• 今後の骨髄線維症治療において、併用療法の役割はどのようになるか

Drawing on the expertise of the world's foremost KOLs, this report provides impartial and balanced insights into the myelofibrosis treatment landscape, highlighting the critical battlegrounds shaping market dynamics. Gain a deeper understanding of current and future treatment paradigms as KOLs critically evaluate the potential of launched and pipeline therapies, offering actionable intelligence to guide strategic decision-making on product positioning and competitiveness.

Key Questions Answered:

• In 2023, the FDA granted orphan drug status Enpaxiq (Pacritinib; CTI Biopharma for use in Myelofibrosis (MF) in patients with platelet counts of below 50 x 109 /L. How has this impacted utilisation?
• How has the use of Jakafi (Ruxolitinib; Incyte Coorporation/Novartis changed over the last 12 months?
• The Phase III FREEDOM2 trial was published in October 2024and reported efficacy gains in MF patients (previously treated with ruxolitinib) vs the best available therapy. What do you see as the impact of this trial on fedrantinib use?
• To what extent do you see Ojjaara replacing ruxolitinib or other therapies for patients with anaemia?
• What do you think is the potential of telomerase inhibitors in the treatment of myelofibrosis? Do you think they offer a good alternative to those patients who have not responded to JAK-inhibitor treatment?
• What are the key unmet needs in the treatment of myelofibrosis? Can artificial intelligence (AI) tools or digital technology address any of these concerns?
• The Phase II SENTRY-2 is assessing efficacy and safety of Xpovio in JAK-inhibitor Naive MF patients, and comparing against approved JAK inhibitors with spleen reduction the primary outcomes. What are your expectations for this trial?
• Syntara released positive data from its Phase II SNT-5505 study, indicating 46% of patients received a 50% decrease in their total system score at 12 weeks, increasing to 80% at 38 weeks How compelling is this data, how would you say this compares to other agents?
• How would you say KER050 compares with similar agents such as luspatercept, which do you expect to be more efficacious?
• What do you think the role of combinations will be in myelofibrosis treatment in the future?

Methodology

Therapy Trend reports are developed through in-depth interviews with the world's foremost Key Opinion Leaders (KOLs) on the current and future treatment landscapes in major disease areas. KOLs are carefully selected based on strict screening criteria, including their global reputation, clinical expertise, and influence within their therapeutic area. Each interview is guided by a meticulously crafted discussion guide. These guides are developed in collaboration with KOLs and peer-reviewed by industry experts to ensure that the questions are comprehensive and pertinent to current market dynamics. Through continuous market monitoring over a 12-month period following the publication of each report, we provide timely updates from KOLs on key news events, market shifts, and clinical developments.

What makes our reports different?

FirstWord Reports, a trusted industry leader with an exclusive focus on the pharmaceutical sector, delivers in-depth, actionable insights for biopharma professionals and decision-makers. Our deep industry knowledge enables us to provide relevant and valuable insights that help you identify emerging trends and navigate complex challenges effectively. Backed by extensive research and independent, impartial insights from leading experts and KOLs, our reports deliver the accuracy and reliability you need. With exclusive access to interviews and data unavailable elsewhere, along with ongoing market monitoring, we give you a comprehensive view of market dynamics. Covering over 40 dynamic disease areas and providing physician intelligence, including KOL insights and quantitative physician surveys, as well as industry expert views on issues in medical affairs, digital health, sales & marketing, market access, and other areas, our reports empower you to make smarter, data-driven decisions and maintain a competitive edge in a fast-moving industry.