Market Challenges And Opportunities

Global Hemoglobinopathy Treatment Drugs Market– Drivers

Increasing research & development activities in hemoglobinopathy treatment

Increasing research & development activities in hemoglobinopathy treatment is expected to drive the global hemoglobinopathy treatment drugs market growth over the forecast period. For instance, on June 16, 2022, researchers at The National Heart, Lung, and Blood Institute (NHLBI), a U.S.-based research organization, announced that activating a protein in red blood cells may improve anemia and alleviate acute episode of severe pain for people living with sickle cell disease. Treatments that stimulate pyruvate kinase (PKR) activity may prevent or alleviate severe pain crisis as PKR deficiency is due to mutation in PKLR gene. A new drug is being develop that can treat patients with inherited pyruvate kinase deficiency. Early phase 1 clinical research trial, showed that activating PKR in patients with SCD through an oral therapy was safe and well tolerated. The researchers will continue to assess the long-term safety and efficacy of this approach with more research. They will specifically look at how this oral therapy may reduce the number of sickling events people experience and also see if it can improve anemia.

Increasing approvals of newer therapies for hemoglobinopathy treatment

Increasing approvals of newer therapies for hemoglobinopathy treatment is expected to drive the global hemoglobinopathy treatment drugs market growth. For instance, on February 17, 2022, Agios Pharmaceuticals, Inc., a U.S.-based pharmaceutical company in the field of cellular metabolism pioneering therapies for genetically defined diseases, announced that the U.S. Food and Drug Administration (FDA) has approved PYRUKYND (mitapivat) in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, lifelong hemolytic anemia. PYRUKYND is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease. The successful ACTIVATE and ACTIVATE-T studies demonstrate the impact of mitapivat in significantly improving hemolysis and anemia in PK deficiency.

Global Hemoglobinopathy Treatment Drugs Market: Restraints

Withdrawal of clinical trials

Withdrawal of clinical trials is expected to hamper the global hemoglobinopathy treatment drugs market growth. For instance, according to clinical trials site clinicaltrials.gov a study was conducted to study peripheral blood stem cell collection from patients with sickle cell disease (scd) using plerixafor. This study was terminated on April 7, 2023 as the safety and efficacy of plerixafor in adult and pediatric SCD patients was an unknown when it was started. Hence recruiting patients without a therapeutic option was not ethically justifiable.

To counterbalance this restraint, proper safety and efficacy evaluation should be carried out to avoid withdrawal of study.

Availability of alternative treatment

Availability of alternative treatment like bone marrow is expected to hamper the global hemoglobinopathy treatment drugs market growth. Bone marrow transplant is only reliable treatment for hemoglobinopathy as most treatment options that are available focus on reducing symptoms of the disorders. For instance, according to the data published by Health Resources and Services Administration, a U.S. government agency, in, June 13, 2023, in 2021, 5,073 unrelated and 4,276 related bone marrow and cord blood transplants were performed in the U.S. The donor registry contains more than 9 million potential donors in the U.S. Nearly 268,000 cord blood units are on the donor registry. This total includes more than 111,000 units from the National Cord Blood Inventory (NCBI), with more than 3,000 NCBI units added in 2022.

To counterbalance this restrain, more awareness on advantages of hemoglobinopathy treatment drugs should be spread.

Increasing research & development activities in hemoglobinopathy treatment
Increasing approvals of newer therapies for hemoglobinopathy treatment

Global Hemoglobinopathy Treatment Drugs Market: Key Developments

On October 18, 2021, Esteve, a Spain-based international pharmaceutical company, announced U.S. Food and Drug Administration (FDA) approved Seglentis (celecoxib and tramadol hydrochloride), a proprietary product developed by Esteve’s R&D team. It is an innovative first-in-class product comprised of a co-crystal form of celecoxib (an anti-inflammatory) and tramadol (an analgesic) for the treatment of acute pain in adults. This is ESTEVE's first proprietary research product to enter the U.S. market. Seglentis is the trade name for tablets that contain a co-crystal composed of celecoxib and tramadol hydrochloride. It is a new analgesic designed for acute pain management in a multimodal treatment approach targeting four complementary pain relief mechanisms. It offers a new treatment option for acute pain management aligned with the multimodal analgesia now considered standard of care.

On March 22, 2021, Pacira BioSciences, Inc., a U.S. based pharmaceutical company, announced the U.S. Food and Drug Administration (FDA) has approved the submission of its supplemental new drug application (sNDA) seeking expansion of the EXPAREL label to include use in patients 6 years of age and older for single-dose infiltration to produce postsurgical analgesia. With this approval, EXPAREL is the first and only FDA approved long-acting local analgesic for the pediatric population as young as age six. The sNDA was based on the positive data from the Phase 3 PLAY study of EXPAREL infiltration in pediatric patients undergoing surgeries. Overall findings were consistent with the pharmacokinetic and safety profiles for adult patients with no safety concerns identified at a dose of 4 mg/kg. The PLAY study enrolled 98 patients to evaluate safety and the pharmacokinetics of EXPAREL for two patient groups: patients aged 12 to less than 17 years and patients aged 6 to less than 12 years. Per U.S. FDA guidance, the primary objectives of the PLAY study were to evaluate the pharmacokinetics and safety of EXPAREL.

On July 1, 2023, India launched national sickle cell anaemia elimination mission aiming to screen more than seven crore young tribals so that the debilitating disease can be detected early and managed with therapies. The mission, announced in the Union budget earlier in 2023, focus on awareness creation and universal screening of people in the 0-40 years age group in tribal areas in 278 districts in 17 states. The government plans to eliminate the disease by 2047. This mission includes distribution of sickle cell genetic status card to beneficiaries that would save lives of children and families affected by disease. Government have also established integrated centers for hemophilia and hemoglobinopathies in 22 tribal districts for treatment and diagnosis of patients.

On June 21, 2022, Precision BioSciences, Inc., a U.S. based biotechnology company, announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis AG, Switzerland based global healthcare company . As part of the Agreement, Precision BioSciences, Inc will develop a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a safe harbor location in the genome as a potential one-time transformative treatment option for diseases including certain hemoglobinopathies such as sickle cell disease and beta thalassemia. While, Novartis AG taking responsibility for all subsequent research, development, manufacturing, and commercialization activities. Precision will receive an upfront payment of US$ 75 million and is eligible to receive up to an aggregate amount of approximately US$ 1.4 billion in additional payments for future milestones.

Global Hemoglobinopathy Treatment Drugs Market: Key Trends

Increasing clinical trials for hemoglobinopathy treatment

Research & development activities for hemoglobinopathy treatment can drive growth of market. On November 6, 2021, Bristol Myers Squibb, a U.S.-based multinational pharmaceutical company, and Acceleron Pharma Inc., a U.S.-based clinical stage biopharmaceutical company, announced the first data from the Phase 2 BEYOND study evaluating Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, plus best supportive care in adult patients with non-transfusion dependent (NTD) beta thalassemia. Results demonstrated that 77.7% of patients treated with Reblozyl achieved a hemoglobin increase (≥1.0 gram/deciliter) compared to 0% of patients in the placebo arm. Changes in patient-reported outcomes also correlated with increases in hemoglobin.

Strategies like agreement by key market players

Strategies like agreement between key market players can drive growth of market. On September 8, 2020, Global Blood Therapeutics, a U.S.-based biopharmaceutical company, announced that it has entered into an exclusive agreement with Biopharma-Middle East and Africa, UAE-based pharmaceutical company to distribute Oxbryta (voxelotor) tablets in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and UAE, collectively known as the Gulf Cooperation Council (GCC) region. There are estimated to be more than 100,000 people age 12 years and older in this region living with sickle cell disease (SCD). Oxbryta is a once-daily, oral therapy that directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD.