A study with 50 U.S. hematologists reveals that approximately one-third have already prescribed Adakveo or Oxbryta
Newton, MA., September 30, 2020 — Sickle cell disease (SCD) is a chronic, progressive condition of genetic origin that affects approximately 100,000 people in the United States. The November 2019 approval of Novartis’ Adakveo (crizanlizumab) and Global Blood Therapeutics’ Oxbryta (voxelotor) by the U.S. Food and Drug Administration expanded the number of approved therapies beyond the standard of care (hydroxyurea) and Emmaus Medical’s Endari. Moreover, an active pipeline of novel gene therapies, innovative small molecule therapeutics, and follow-on agents from Novartis and Global Blood Therapeutics signals a market that will become increasingly complex and fragmented.
According to a Q3 study from REACH Market Research included in its PhysiciansTreating®* research service, an estimated 6% of SCD patients have been prescribed Adakveo or Oxbryta since the beginning of the year with Oxbryta edging out Adakveo in terms of the number of drug-treated patients. The study also finds that a patient’s predominant clinical phenotype is a key driver in hematologists’ decisions on which of these two new therapies to prescribe. In fact, 85% of Adakveo prescribers use the drug in patients where the predominant clinical phenotype is frequent vaso-occlusive crises (VOCs) where as Oxbryta’s use is broad including transfusion-dependent patients, patients with hemoglobin levels less than 10 g/dL, patients who have failed hydroxyurea, and patients with VOCs.
“Adakveo and Oxbryta have added a new level of complexity to the SCD treatment algorithm and brought new hope for SCD patients. However, there is so much more work to be done in the area of therapeutics given that patients still have significant clinical unmet needs. The most pressing need is for novel therapies that can modify the course of the disease by slowing down and ultimately halting end organ damage.” said President and Founder of REACH, Danielle Drayton, PhD.
Among eleven unmet needs identified in this study, four areas emerged as “extremely” important by a majority of hematologists – slow progression of end organ damage, reduce the incidence of acute chest syndrome in pediatric patients, reduce hospitalizations for SCD-related pain, and reduce occurrence of painful vaso-occlusive crises. These unmet needs represent significant gaps in the market and shed light on the opportunity for the newest approved therapies as well as pipeline assets to fill these voids.
“This is a fascinating market with many parallels to the trajectories of the HIV and Hepatitis C Virus therapeutic markets, including treatment evolution to more disease-modifying options and the use of combination therapy. Today the focus of current and emerging SCD therapies is on monotherapy. Going forward, we fully expect to see combination therapy with and without hydroxyurea to be an important next phase of the treatment evolution” said Dr. Drayton.
While the pipeline for SCD is fairly active with at least 20 novel therapies, unaided awareness of gene therapy (gene addition and gene editing) dwarfs awareness of all other pipeline therapies. Among the 40% of surveyed hematologists who have any unaided awareness of pipeline drugs, 75% mentioned gene therapy. More importantly, hematologists estimate that as many as one-third of their SCD patients would be candidates for gene therapy although they are split on the clinical phenotype of gene therapy candidates. REACH will continue to monitor and analyze the impact of Adakveo and Oxbryta on SCD medical practice, changes in the patient’s treatment journey and the impact of clinical, commercial, and regulatory events on the overall market.
About PhysiciansTreating®
PhysiciansTreating® combines quantitative and qualitative primary market research with specialists to understand current treatment and disease management practices as well as anticipated changes. These syndicated studies provide analysis of physicians’ attitudes, perceptions, and self-reported prescribing behavior and evaluate perceived product advantages and disadvantages.
Other Sickle Cell Disease market research studies include the next wave of PhysiciansTreating® U.S. (Q4 2020), PatientsWith®, and Market Insights 360. PatientsWith® Sickle Cell Disease provides insight into the patient’s experience with their disease and treatment. PatientsWith® also uncovers patients’ knowledge and perceptions of their disease and how they seek out disease and treatment-related information. MarketInsights 360 combines primary and secondary market research to analyze the competitive landscape, market dynamics, value and access considerations, and future trends that will shape how SCD patients are treated.
About REACH Market Research
REACH is an independent pharmaceutical market research company focused on rare diseases and orphan drugs. Our work combines patient and physician research with disease market understanding to improve decision-making for early-stage pipeline assets. We help life sciences companies deepen understanding of the patient and physician journey, determine the clinical and commercial viability of early-stage assets, understand current and future treatment paradigms, and assess the impact of market and competitor dynamics.
For more information contact: info@reachmr.com
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*PhysiciansTreating trademark is pending