In an August 3rd, 2022 news release from Syros Pharmaceuticals (“Syros”), the company shared that its product tamibarotene had received a positive opinion for its Orphan Drug designation application. Tamibarotene is designed for patients with myelodysplastic syndromes (MDS). 

Syros explains that tamibarotene is:

a selective retinoic acid receptor alpha (RARα) agonist that we are developing for genomically defined subsets of patients whose disease is characterized by the overexpression of the RARA gene.

This treatment option is orally administered. Currently, Syros is evaluating tamibarotene – in conjunction with azacitidine – within the Phase 3 SELECT-MDS-1 study. Additional research is evaluating the therapy for patients with acute myeloid leukemia (AML).

In the European Union (EU), Orphan Drug designation is granted following or based upon a positive EMA opinion. As the EMA explains:

to qualify for orphan designation, a medicine must meet a number of criteria:

• It must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
• The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that the marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
• No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.” 

What are Myelodysplastic Syndromes (MDS)? 

Myelodysplastic syndromes (MDS) are a group of progressive conditions which prevent the bone marrow from producing enough healthy platelets, red blood cells, and white blood cells. Instead, the bone marrow produces immature cells which fail to mature or never leave the bone marrow. Doctors and scientists are unsure of what causes MDS. However, many hypothesize that people have a genetic predisposition to MDS which may be triggered by chemical exposure, chemotherapy, or radiation. MDS occurs most often in individuals aged 60+, and more often in males than females. In around 50% of diagnoses, MDS progresses to become AML. Symptoms can (but do not always) include:

• Anemia (low red blood cell count)
• Neutropenia (low white blood cell count)
• Thrombocytopenia (low platelet count)
• Fatigue and general malaise
• Heart palpitations
• Easy bruising and bleeding
• Frequent infections
• Shortness of breath
• Extremely pale skin
• Chest pain