

Drug discovery
30
drugs
With orphan designations
Overview
Precursor B-cell acute lymphoblastic leukemia (B-ALL) is an aggressive hematologic malignancy characterized by uncontrolled proliferation of immature B-cell lymphoblasts in bone marrow and blood. It accounts for 75–80% of pediatric ALL cases and 75% of adult ALL cases [1][6]. Prognosis depends on age, cytogenetics (e.g., BCR-ABL1, ETV6-RUNX1), and treatment response, with 5-year survival rates exceeding 90% in children but ≤60% in adults [12][16]. Modern protocols combine chemotherapy, targeted therapies (e.g., blinatumomab, TKIs), and allogeneic hematopoietic cell transplantation (allo-HCT) for high-risk or relapsed cases [3][8].
Therapies
First-line: Pediatric/adolescent regimens (BFM-based protocols) yield 85–95% complete remission [3][12]
Relapsed/refractory: Immunotherapy (blinatumomab/inotuzumab) followed by allo-HCT doubles survival vs standard chemotherapy [3][14]
Emerging approaches: CAR-T cell therapy for multi-relapsed cases and MRD-directed therapies [13][18]
Categories: rare hematological diseases, rare neoplastic diseases, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
enzomenib | small molecules | FDA | 2026-07-19 | — | Sumitomo Pharma America, Inc. |
cell therapy product derived from an allogeneic hematopoietic stem cell donor and expressed anti-CD19 chimeric antigen receptor, T cell receptor of invariant natural killer T cells, and interleukin-15 | cell therapies | FDA | 2026-04-16 | — | Shanghai Allovanta Biotechnology Co., Ltd. |
blinatumomab | antibodies | FDA | 2025-12-12 | — | Amgen Inc. |
surovatamig | antibodies | FDA | 2025-11-09 | — | AstraZeneca Pharmaceuticals LP |
universal chimeric antigen receptor T cells (UCAR-T) targeting CD7 | cell therapies | FDA | 2025-11-04 | — | Bioheng Therapeutics US LLC |
chimeric antigen receptor T cells targeting CD19 & CD22 | cell therapies | FDA | 2025-08-19 | — | Shenzhen Pregene Biopharma Co., Ltd. |
Allogeneic peripheral blood-derived T-cells, fratricide-resistant, transduced with a lentivirus vector expressing a chimeric antigen receptor against CD7 | cell therapies | EMA | 2025-07-18 | — | Yes Pharmaceutical Development Services GmbH |
small molecule cytidine triphosphate synthase 1 (CTPS1) inhibitor | small molecules | FDA | 2025-05-02 | — | Step Pharma |
adeno-associated viral serotype rh74 vector encoding an anti-CD19-anti-CD3 bispecific protein | gene therapies | FDA | 2024-09-13 | — | Vironexis Biotherapeutics, Inc. |
Anti-CD19 Chimeric Antigen Receptor T-Cell Injection | cell therapies | FDA | 2022-01-13 | — | Juventas Cell Therapy Ltd. |
Axicabtagene ciloleucel | cell therapies | EMA | 2015-11-11 | — | Kite Pharma EU B.V. |
Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 | cell therapies | EMA | 2015-08-10 | — | Fate Therapeutics Limited |
Spanlecortemlocel | cell therapies | EMA | 2014-12-16 | — | Novartis Europharm Limited |
Pinometostat | small molecules | EMA | 2014-01-16 | — | Voisin Consulting Life Sciences |
Moxetumomab pasudotox | proteins | EMA | 2013-07-17 | — | AstraZeneca AB |
Inotuzumab ozogamicin [Besponsa] | antibodies | EMA | 2013-06-07 | 2017-07-03 | Pfizer Europe MA EEIG |
inotuzumab ozogamicin [Besponsa] | antibodies | FDA | 2013-03-25 | 2024-03-06 | Wyeth Pharmaceuticals, LLC |
inotuzumab ozogamicin | antibodies | FDA | 2013-03-25 | 2017-08-17 | Wyeth Pharmaceuticals, LLC |
inotuzumab ozogamicin [Besponsa] | antibodies | FDA | 2013-03-25 | 2024-03-06 | Wyeth Pharmaceuticals, LLC |
Ponatinib hydrochloride [Iclusig] | small molecules | EMA | 2010-02-02 | — | Incyte Biosciences Distribution B.V. |
Mercaptopurine [Xaluprine] | small molecules | EMA | 2009-04-30 | — | Nova Laboratories Ireland Limited |
Allogeneic umbilical cord blood cells-derived CD133+ cells ex vivo expanded | cell therapies | EMA | 2009-02-27 | — | Regulatory Resources Group Limited |
Vincristine sulfate | small molecules | EMA | 2008-07-08 | — | Pharma Gateway AB |
Dasatinib [Sprycel] | small molecules | EMA | 2005-12-23 | — | [INACTIVE] Bristol-Myers Squibb Pharma EEIG |
Imatinib mesilate [Glivec] | small molecules | EMA | 2005-08-26 | — | Novartis Europharm Limited |
Nelarabine [Atriance] | small molecules | EMA | 2005-06-16 | — | Novartis Europharm Limited |
Asparaginase [Spectrila] | proteins | EMA | 2005-01-26 | — | [INACTIVE] Medac Gesellschaft fuer klinische Spezialpraeparate mbH |
Plitidepsin [Aplidin] | small molecules | EMA | 2003-07-09 | — | Pharma Mar S.A. |
Clofarabine [Evoltra] | small molecules | EMA | 2002-02-05 | — | [INACTIVE] Sanofi B.V. |
Ricin (blocked) conjugated murine mca (anti-b4) | antibodies | FDA | 1991-01-24 | — | ImmunoGen, Inc. |