Drug discovery
67
drugs
With orphan designations
Overview
Follicular Lymphoma (FL) is an indolent B-cell non-Hodgkin lymphoma characterized by slow progression, frequent relapses, and incurability. It primarily affects older adults (median age 60–65) and is often diagnosed at advanced stages (III/IV). FL involves CD20+ B-cells with chromosomal t(14;18) translocations in 85% of cases, driving BCL2 overexpression [1][7][12]. Treatment strategies range from watchful waiting for asymptomatic patients to chemoimmunotherapy, targeted therapies (e.g., PI3K/EZH2 inhibitors), and emerging immunotherapies (e.g., CAR-T) [3][5][8]. The 5-year relative survival is 89.9%, but disease burden escalates with later treatment lines [2][4][9].
Burden
Clinical: 34% mortality at 3-year follow-up for ≥3L therapy; 20% experience early progression (POD24) [4][9][14].
Treatment lines: 66% receive ≥2 lines, 34% ≥3 lines over 20 years [9][19].
Economic: Mean annual costs rise to €22,230/patient with later lines, driven by hospitalizations and novel therapies [4][14].
Therapies
Categories: rare hematological diseases, rare neoplastic diseases, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
microbiome-based therapeutic peptide cancer immunotherapy composed of 5 synthetic peptides | peptides | FDA | 2026-05-20 | — | Enterome SA |
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 | 2026-03-02 | — | Shanghai Allovanta Biotechnology Co., Ltd. |
surovatamig | antibodies | FDA | 2026-02-20 | — | AstraZeneca Pharmaceuticals LP |
small molecule bifunctional cereblon-dependent ligand directed degrader (LDD) of BCL6 | small molecules | FDA | 2026-02-13 | — | Bristol Myers Squibb |
Surovatamig | antibodies | EMA | 2026-01-09 | — | AstraZeneca AB |
allogeneic CRISPR/Cas9-mediated genetically modified CAR T cells targeting CD19 antigen | cell therapies | FDA | 2025-04-04 | — | CRISPR Therapeutics, Inc. |
Tafasitamab [Minjuvi] | antibodies | EMA | 2025-02-26 | 2025-12-16 | Incyte Biosciences Distribution B.V. |
orally available BTK-targeting chimeric degradation activation compound designed to degrade wildtype BTK and multiple mutant forms | small molecules | FDA | 2024-12-17 | — | BeOne Medicines USA, Inc. |
recombinant humanized anti-CD3/CD19 bispecific antibody | antibodies | FDA | 2024-04-22 | — | Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. |
Golcadomide hydrochloride | small molecules | EMA | 2023-12-13 | — | Bristol-Myers Squibb Pharma EEIG |
2-[(3S)-2,6-dioxopiperidin-3-yl]-4-{[(2-fluoro-4-{[3-(morpholin-4-yl)azetidin-1-yl]methyl} phenyl) methyl]amino}-1H-isoindole-1,3(2H)-dione-hydrogen chloride (1/1) | small molecules | FDA | 2023-01-25 | — | Celgene Coporation |
zanubrutinib [Brukinsa] | small molecules | FDA | 2022-11-08 | 2024-03-07 | BeOne Medicines USA, Inc. |
Imvotamab | antibodies | FDA | 2022-10-31 | — | IGM Biosciences, Inc. |
Edited allogeneic chimeric antigen receptor T Cells (CAR-T) | cell therapies | FDA | 2022-09-01 | — | Caribou Biosciences, Inc. |
Odronextamab [Ordspono] | antibodies | EMA | 2022-07-18 | — | Regeneron Ireland Designated Activity Company |
Epcoritamab [Tepkinly] | antibodies | EMA | 2022-06-21 | — | Abbvie Deutschland GmbH & Co. KG |
epcoritamab-bysp [Epkinly] | antibodies | FDA | 2022-03-01 | 2024-06-26 | Genmab US, Inc. |
Anti- FcgammaRIIB Antibody | antibodies | FDA | 2022-01-18 | — | BioInvent International AB |
Mosunetuzumab [Lunsumio] | antibodies | EMA | 2021-11-12 | 2022-06-07 | Roche Registration GmbH |
Zandelisib | small molecules | FDA | 2021-11-09 | — | MEI Pharma, Inc. |
Tisagenlecleucel [Kymriah] | cell therapies | EMA | 2021-07-19 | 2022-05-03 | Novartis Europharm Limited |
an autologous Chimeric Antigen Receptors (CAR) T-cell therapy expressing CD19/CD20 bi-specific CAR | cell therapies | FDA | 2021-05-17 | — | Janssen Research & Development, LLC |
4-{4-{[6-(4-Chlorophenyl)spiro[3.5]non-6-en-7-yl]methyl}-piperazin-1-yl}-N-{{3-nitro-4-[((2S)-1,4-dioxan-2-ylmethyl)amino]phenyl}sulfonyl}-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide | small molecules | FDA | 2021-01-19 | — | Ascentage Pharma Group Inc. |
tafasitamab-cxix [Monjuvi] | antibodies | FDA | 2021-01-07 | 2025-06-18 | Incyte Corporation |
tisagenlecleucel [Kymriah] | cell therapies | FDA | 2020-09-16 | 2022-05-27 | Novartis Pharmaceuticals Corporation |
umbralisib [Ukoniq] | small molecules | FDA | 2020-03-04 | 2021-02-05 | TG Therapeutics, Inc. |
Parsaclisib | small molecules | FDA | 2019-09-25 | — | Incyte Corporation |
apilimod dimesylate | small molecules | FDA | 2019-07-01 | — | AI Therapeutics, Inc. |
6-{[(1R,2S)-2-aminocyclohexyl]amino-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate | small molecules | FDA | 2019-02-25 | — | Wells Therapeutics, Inc. |
mosunetuzumab-axgb [Lunsumio] | antibodies | FDA | 2018-12-17 | 2022-12-22 | Genentech, Inc. |
mosunetuzumab-axgb [Lunsumio Velo] | antibodies | FDA | 2018-12-17 | 2025-12-19 | Genentech, Inc. |
N-[5-(6-fluoro-8-{[4-(2-hydroxypropan-2-yl)piperidin-1-yl]methyl}-2-morpholinoquinazolin-4-yl)-2-methoxypyridin-3-yl]methanesulfonamide | small molecules | FDA | 2018-10-10 | — | Shanghai Yingli Pharmaceutical Co., Ltd. |
Lisocabtagene maraleucel [Breyanzi] | cell therapies | EMA | 2018-05-25 | — | Bristol-Myers Squibb Pharma EEIG |
Tazemetostat | small molecules | EMA | 2018-03-21 | — | Ipsen Pharma |
avadomide | small molecules | FDA | 2018-03-12 | — | Celgene, a wholly owned subsidiary of Bristol-Myers Squibb |
humanized IgG4-based anti-CD20 x anti-CD3 bispecific monoclonal antibody | antibodies | FDA | 2018-01-25 | — | Regeneron Pharmaceuticals, Inc. |
tazemetostat [Tazverik] | small molecules | FDA | 2017-11-14 | 2020-06-18 | Epizyme, Inc. |
GLUCOPYRANOSYL LIPID A | small molecules | EMA | 2017-10-13 | — | [INACTIVE] Immune Design Limited |
lisocabtagene maraleucel [Breyanzi] | cell therapies | FDA | 2017-09-07 | 2021-02-05 | Juno Therapeutics, Inc., a Bristol-Myers Squibb Company |
pembrolizumab | antibodies | FDA | 2017-06-06 | — | Merck, Sharp & Dohme Corp |
Glucopyranosyl lipid A stable emulsion | small molecules | FDA | 2017-02-14 | — | Immune Design Corp. |
rituximab and recombinant human hyaluronidase [Rituxan SC] | antibodies | FDA | 2016-08-22 | 2017-06-22 | Genentech, Inc. |
axicabtagene ciloleucel [Yescarta] | cell therapies | FDA | 2016-04-25 | 2017-10-18 | Kite Pharma, Inc. |
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor [Yescarta] | cell therapies | EMA | 2015-11-11 | 2022-06-23 | Kite Pharma EU B.V. |
daratumumab | antibodies | FDA | 2015-08-06 | — | Janssen Research & Development, LLC |
Obinutuzumab [Gazyvaro] | antibodies | EMA | 2015-06-19 | 2016-06-15 | Roche Registration GmbH |
obinutuzumab [GAZYVA] | antibodies | FDA | 2015-04-15 | 2016-02-26 | Genentech Inc., a member of the Roche Group |
copanlisib [ALIQOPA] | small molecules | FDA | 2015-02-05 | 2017-09-14 | Bayer US LLC |
ibrutinib | small molecules | FDA | 2014-09-08 | — | Pharmacyclics, LLC |
177Lu-tetraxetan-tetulomab | antibodies | EMA | 2014-06-04 | — | Nordic Nanovector AS |
177Lu-tetraxetan-tetulomab | antibodies | FDA | 2014-05-14 | — | Nordic Nanovector AS |
Ibrutinib [Imbruvica] | small molecules | EMA | 2013-12-18 | — | Janssen Cilag International |
idelalisib [Zydelig] | small molecules | FDA | 2013-09-26 | 2014-07-23 | Gilead Sciences, Inc. |
lenalidomide [Revlimid] | small molecules | FDA | 2013-09-17 | 2019-05-28 | Celgene Corporation |
duvelisib [COPIKTRA] | small molecules | FDA | 2013-08-01 | 2018-09-24 | Secura Bio, Inc. |
Duvelisib [Copiktra] | small molecules | EMA | 2013-07-17 | — | [INACTIVE] Verastem Europe GmbH |
Idelalisib | small molecules | EMA | 2013-07-17 | — | Gilead Sciences International Limited |
Lenalidomide [Revlimid] | small molecules | EMA | 2013-01-24 | — | Celgene Europe B.V. |
humanized IgG1 monoclonal anti-CD20 antibody | antibodies | FDA | 2011-05-26 | — | MENTRIK Biotech, LLC |
bortezomib | small molecules | FDA | 2011-01-04 | — | Millennium Pharmaceuticals, Inc. |
dasiprotimut-T | vaccines | FDA | 2009-10-28 | — | Biovest International, Inc. |
4, 5 dibromorhodamine methyl ester | small molecules | FDA | 2008-10-30 | — | Kiadis Pharma Netherlands B.V. |
pralatrexate | small molecules | FDA | 2008-10-20 | — | Acrotech Biopharma LLC |
Autologous tumor-derived immunoglobulin idiotype coupled to keyhole limpet haemocyanin | proteins | EMA | 2006-08-28 | — | Biovest Europe Limited |
Recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors | proteins | EMA | 2004-12-23 | — | CellGenix GmbH |
IODINE (131I) TOSITUMOMAB [Beximab] | other | EMA | 2003-02-14 | — | Glaxosmithkline Research & Development Limited |
IODINE (131I) TOSITUMOMAB [Beximab] | antibodies | EMA | 2003-02-14 | — | Glaxosmithkline Research & Development Limited |