The Acute Myeloid Leukemia (AML) market is estimated to be valued at USD 1.613 billion in 2024 and is expected to reach USD 2.68 billion by 2031, growing at a compound annual growth rate (CAGR) of 7.5% from 2024 to 2031.

The growing geriatric population who are susceptible to develop AML combined with the rising demand for targeted drug therapy for better treatment outcomes are key factors expected to drive the growth of the AML market.

Market Driver - Increased Incidence of AML due to an Aging Population

As the population ages worldwide, incidences of acute myeloid leukemia are expected to rise substantially in the coming years. It is a well-established fact that AML affects adults with around 70% of cases being diagnosed in people aged above 65 years.

It is projected that number of people aged 65 years or older globally will double from present levels to over 1.5 billion by 2050. People in this age bracket have considerably higher chances of contracting AML compared to younger individuals. With more of the population entering the high-risk age brackets, absolute numbers of AML cases cannot remain unchanged. Even in developed markets where healthcare systems are well equipped, higher caseload will put pressure on existing resources and infrastructure.

Declining immune system and increased susceptibility to DNA damage can make elderly more prone to blood cancers like AML. Higher prevalence of comorbidities also contributes to poorer outcomes in older patients. While R&D efforts continue for developing safer and more effective treatment options, rising geriatric population alone guarantees rising AML incidence rates in future.

Even if cure rates improve marginally, the anticipated spike in older demographic will outweigh such gains, leading to a net increase in AML patient numbers requiring care.

Market Driver - Advancements in Targeted Therapies and Personalized Medicine

The oncology field has seen transformative research in targeted therapies and precision medicine approaches for blood cancers in last decade. More sophisticated understanding of molecular pathogenesis at genetic level has enabled development of novel treatment regimens tailored to individual patient's disease profile. This shift towards biomarker-driven personalized care holds promising prospects for improving AML management.

With the advent of genomic profiling technologies, clinicians are now able to identify specific genetic mutations and abnormalities driving a patient's leukemia. Based on such molecular insights, targeted drugs blocking critical pathogenetic pathways and proteins can be selected. Several targeted agents attacking mutations in genes like FLT3, IDH1/2, BCL-2 have demonstrated clinically meaningful responses in clinical trials. Other targeted classes under investigation includes tyrosine kinase inhibitors, hypomethylating agents, BCL-2 inhibitors and immunotherapy agents.

Furthermore, adaptive trial designs are being increasingly used to fast-track regulatory approval of new therapies shown to be effective for certain biomarker-defined subsets. This allows quicker access to custom treatments. Combinatorial regimens employing multiple targeted drugs also hold potential to achieve improved remission rates by simultaneously blocking multiple cancer growth pathways. Overall, technological evolution provides much optimism regarding improved outcomes, increased survival times and higher quality of life for AML patients.

Market Challenge - High Treatment Costs Limiting Access in Low-income Regions

At nearly $150,000 per patient in the United States, the high costs of AML treatment pose a significant challenge for access in lower-income regions globally. Most AML drugs require lengthy hospital stays for intensive chemotherapy administration and many patients also require bone marrow transplantation, further increasing costs.

In developing countries and emerging markets, healthcare budgets are much more constrained and standard AML regimens may exceed what public insurance or out-of-pocket payments can support. This creates inequity where patients in wealthy countries have access to the latest drugs while patients elsewhere face worse outcomes due only to financial limitations.

Addressing these access issues through efforts like tiered pricing models or partnerships with global health organizations could help expand availability of novel agents in regions where costs serve as the primary barrier to treatment.

Market Opportunity - Development of Novel Therapies with Better Safety Profiles

There is potential for novel AML therapies to not only improve survival rates but also address some of the challenges associated with existing regimens' adverse side effects and tolerability issues. Areas of exploration include targeted therapies that interfere with specific molecular pathways driving leukemic cells or immunotherapies that harness the body's own immune system.

A promising new class is antibody drug conjugates that home payload anticancer drugs directly to leukemia cells. Development of therapies with enhanced safety profiles could make treatments more suitable for older or sicker patient populations and avoid hospitalizations for chemotherapy administration. This could lower the overall cost of care while broadening the markets accessible to novel agents.

Acute Myeloid Leukemia (AML) is typically treated through induction chemotherapy, aimed at putting the disease into remission. The frontline treatment for younger patients involves a "7+3" regimen - 7 days of the chemotherapeutic agent cytosine arabinoside (brand name: Cytosar-U) combined with 3 days of an anthracycline such as daunorubicin (brand name: Cerubidine) or idarubicin (brand name: Zavedos).

For older or unfit patients where a "7+3" is considered too toxic, lower intensity options include low-dose cytarabine alone or in combination with hypomethylating agents like azacitidine (brand name: Vidaza) or decitabine (brand name: Dacogen). Post-remission treatment then involves consolidation chemotherapy using high-dose cytarabine, with or without an anthracycline, or a hematopoietic stem cell transplant if a suitable donor is available.

For relapsed or refractory disease, physicians generally turn to experimental therapies first. Venetoclax (brand name: Venclexta) combined with low-dose cytarabine is a popular option for older patients unable to tolerate intensive therapy. Younger patients may receive salvage chemotherapy with mitoxantrone, etoposide, and cytarabine or clofarabine before proceeding to a stem cell transplant if possible.

Key factors influencing prescribers' preferences include the patient's age, performance status, comorbidities, and genetic/molecular risk factors which determine the aggressiveness of treatment. Insurance coverage and cost are also growing considerations.

AML can be broadly classified into standard-risk and high-risk disease based on cytogenetics and molecular abnormalities. Treatment involves induction chemotherapy in both risk groups with the goal of achieving remission.

For younger fit patients (<60yrs) with standard-risk AML, the standard first-line treatment is a "7+3" regimen comprising continuous infusion of cytarabine (Ara-C) for 7 days along with an anthracycline like daunorubicin for 3 days. This induces remission in 70-80% of patients. Post-remission, consolidation chemotherapy with high-dose Ara-C is given to prevent relapses.

For high-risk AML or relapsed/refractory disease, the preferred regimen involves an anthracycline like idarubicin or mitoxantrone instead of daunorubicin for a "3+7" schedule, with midostaurin or gemtuzumab ozogamicin added for patients with FLT3 mutations. Stem cell transplantation, if possible, offers the best chance of cure for such patients in first remission.

For relapsed/refractory AML ineligible for transplantation, venetoclax-based combination therapies have shown promising results. Venetoclax when combined with low-dose Ara-C led to a complete remission rate of 54% and median overall survival of 14.7 months in this high-risk population. Kindler et al established this novel regimen as an effective salvage treatment option.

One of the most successful strategies adopted was developing targeted therapies with novel mechanisms of action. In 2017, Pfizer's drug Rydapt (midostaurin) was approved for FLT3 mutant AML. It targets FLT3 mutations, present in around 30% of AML patients. Rydapt was the first drug approved specifically for FLT3 mutant AML. It helped improve survival rates and is still a standard treatment option.

Another example is GSK's Tafinlar + Mekinist combination, approved in 2018. It specifically targets mutations in genes like KIT and RAS. Prior to this, there were no targeted therapies available for these mutations. This expanded treatment options for patients with certain high-risk genetic subtypes.

Celgene's Vidaza and Dacogen gained approval in 2004 and 2006 respectively as the first drugs in over 30 years for AML. They helped reshape standard of care at the time by improving symptoms and survival. This established hypomethylating agents as a mainstay in AML therapy.

More recently in 2017, Pfizer's BCL-2 inhibitor Venclexta gained accelerated approval in combination with azacitidine or decitabine or low-dose cytarabine for older AML patients. This marked the first approval of a BCL-2 inhibitor and expanded options. In trials, it helped increase complete response rates and overall survival periods.

Insights, By Therapy: The Dominance of Established Drugs in Treatment Drives Chemotherapy's High Share

In terms of therapy, chemotherapy contributes the highest share of the market owning to the availability of several established drug options that have been the mainstay of AML treatment. Chemotherapy takes advantage of the fact that AML is a cancer of the blood and bone marrow, with malignant cells that divide more rapidly than normal cells.

Chemotherapeutic drugs work by killing rapidly dividing cancer cells through various mechanisms. Drugs like cytarabine, anthracyclines like daunorubicin and idarubicin, and hypomethylating agents like azacitidine are highly effective at inducing remission in AML patients and have decades of clinical evidence establishing their efficacy and safety profiles.

The wide acceptance and familiarity of oncologists and patients with these drugs contributes to chemotherapy remaining the treatment of choice, especially in newly diagnosed and elderly patients.

Insights, By Route of Administration: Oral Drugs Lead Growth in Route of Administration

In terms of route of administration, oral contributes the highest share of the market driven by growing acceptance of novel oral drugs. Several novel AML drugs recently approved or in late stages of development are administered orally rather than intravenously like traditional chemotherapy.

Drugs like the BCL-2 inhibitor venetoclax and FLT3 inhibitor gilteritinib have proven efficacy when given as oral formulations. This provides distinct advantages of convenience, cost savings from reducing hospital visits and outpatient drug administration. Oral administration improves treatment adherence and compliance too.

Growth in research into oral formulations will likely see this segment dominate the route of administration for AML treatment going forward.

Insights, By End User: Hospitals Corner Major Disease Management Due to Infrastructure

In terms of end user, hospitals contribute the highest share of the AML market owing to their advanced infrastructure and resources for cancer management. AML diagnosis and treatment often involves complex regimens like induction, consolidation and maintenance chemotherapy requiring inpatient hospital stays.

Hospitals have dedicated oncology departments, bone marrow transplant facilities and well-trained physicians and nurses familiar with intensive chemotherapy protocols. Managing adverse effects from therapies also benefits from the round-the-clock monitoring hospitals provide.

While specialty clinics are increasingly involved in long-term follow ups, hospitals remain the primary point of care for initial management, induction therapy and subsequent lines of treatment for AML. This drives their position as the major end-user segment.

• Key data highlights the 42,000+ incident cases of AML in major markets in 2022, with this number expected to reach around 48,000 by 2031. The high prevalence of AML in the U.S. and Japan is driving market growth​.
• The increase in collaborations between pharmaceutical companies for rare disease research has accelerated progress in treatment options, especially in the areas of gene therapy and RNA-based solutions.

The major players operating in the Acute Myeloid Leukemia (AML) Market include Bristol-Myers Squibb, AbbVie, Pfizer, F. Hoffmann-La Roche, and Novartis.

• In 2022, the FDA approved REZLIDHIA™ (olutasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a susceptible IDH1 mutation. Olutasidenib specifically targets mutations in the isocitrate dehydrogenase-1 (IDH1) gene, which is implicated in a subset of AML cases. This approval expands therapeutic options for patients with relapsed or refractory AML, offering a targeted treatment for those with this genetic mutation, enhancing the overall management of the disease.
• Bristol-Myers Squibb (BMS) announced that it has been making progress in its pipeline for Acute Myeloid Leukemia (AML) therapies, particularly in the area of combination therapies. BMS focuses on advancing innovative treatments by combining its existing drugs and investigational agents to improve patient outcomes. The company's research in oncology, including AML, explores how combination therapies can provide more effective treatment options, particularly for challenging cancer types like AML, which has traditionally been difficult to treat with a single drug approach.

• By Therapy
  • Chemotherapy
  • Targeted Therapy
  • Immunotherapy
• By Route of Administration
  • Oral
  • Intravenous (IV)
• By End User
  • Hospitals
  • Specialty Clinics
• By Distribution Channel
  • Hospital Pharmacy
  • Retail Pharmacy