이 보고서는 세계 최고의 KOL의 전문성을 바탕으로 골수섬유증 치료 전망에 대한 공정하고 균형 잡힌 통찰력을 제공하고, 시장 역학을 형성하는 주요 쟁점에 초점을 맞춥니다. 제품 포지셔닝과 경쟁에 대한 전략적 의사결정에 도움이 되는 실용적인 정보를 제공함으로써 현재와 미래의 치료 패러다임을 더 깊이 이해할 수 있도록 돕습니다.
보고서 내용
2023년 FDA, 혈소판 수 50x109/L 미만의 골수섬유증(MF) 환자에 대한 Enpaxiq (Pacritinib, CTI Biopharma)의 희귀의약품(희귀질환 치료제) 사용 승인은 치료에 어떤 영향을 미칠까?
Jakafi(Ruxolitinib, Incyte Corporation/Novartis)의 지난 12개월간 사용량 변화 추이
2024년 10월에 발표된 FREEDOM2 임상 3상 시험에서 MF 환자(Ruxolitinib 이전 치료 경험이 있는 환자)에서 사용 가능한 최선의 치료법 대비 효능이 개선된 것으로 보고된 가운데, 이 시험이 페드란티닙 사용에 어떤 영향을 미칠 것인가?
Ojjaara가 빈혈 환자에게 ruxolitinib이나 다른 치료제를 대체할 수 있는 가능성은 어느 정도인가?
골수섬유증 치료에서 텔로머라아제 억제제의 가능성과 JAK 억제제 치료에 반응하지 않는 환자에게 텔로머라아제 억제제가 좋은 선택이 될 수 있는가?
골수섬유증 치료에서 미충족 수요는 무엇이며, 인공지능(AI) 도구와 디지털 기술이 이러한 우려에 대응할 수 있을까?
임상 2상인 SENTRY-2는 JAK 억제제 순응군 골수섬유증 환자를 대상으로 Xpovio의 유효성과 안전성을 평가하고, 비장 축소를 주요 결과로 승인된 JAK 억제제와 비교하는 임상 2상 시험 중인데, 이 시험에 대한 기대는 무엇인가?
Syntara는 2상 SNT-5505 연구에서 긍정적인 데이터를 발표했는데, 12주째에 환자의 46%가 총 시스템 점수가 50% 감소했으며 38주째에는 80%로 증가했다는 결과가 나왔다. 이 데이터가 얼마나 설득력이 있으며, 다른 약제와 비교하면 어떠한가?
luspatercept와 같은 유사 약물과 비교했을 때 KER050의 효능은 어느 쪽이 더 우수할 것으로 예상되는가?
향후 골수섬유증 치료에서 병용요법의 역할은 어떻게 될 것인가?
LSH
영문 목차
영문목차
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.
