CellCentric, a UK-based biotechnology company, has announced that the US Food and Drug Administration (FDA) has granted it the orphan drug designation for inobrodib in the treatment of multiple myeloma.
Delivered as an oral capsule, inobrodib can be used at home without the need for intensive monitoring. This is a first-class drug, with a new mechanism of action. It can be taken by patients who are unresponsive to other treatments, and in combination with existing standard care drugs.
Orphan drug status granted by the FDA to support drug development for disorders affecting fewer than 200,000 people in the U.S. The benefits of designating orphan drugs can include market exclusivity for up to seven years after regulatory approval, waiver of FDA application fees, and tax credits for qualifying clinical trials. .
The ongoing CellCentric blood cancer trial is enrolling patients with a variety of haematological malignancies including multiple myeloma, but also non-Hodgkin lymphoma, acute myeloid leukemia, and high risk myelodysplastic syndrome (MDS).
CellCentric plans to announce its expanding cohort data at the 65th ASH Annual Meeting and Exposition in San Diego in December. This will include data from trials of inobrodib to treat multiple myeloma either as monotherapy or in combination with pomalidomide and dexamethasone.
“The designation of the orphan drug for inobrodib is an important milestone in our journey to develop additional therapeutic options for patients with multiple myeloma,” said Tomasz Knurowski, chief medical officer of CellCentric.
“We look forward to providing an update on our clinical data by the end of the year, which will help inform the next stages of inobrodib development.”
Inobrodib works by binding to certain parts of the p300 protein and CBP (bromodomain). It is highly selective and does not affect similar pockets in other proteins. Through this action inobrodib reduces the expression of the main cancer triggers: IRF4, MYC, and the androgen receptor (AR and its variants).
This is a new type of treatment for cancer patients. As an oral capsule, it is easier for the patient and can be taken home without the need for intensive monitoring. Because it has a good safety profile for the drug in this setting, it can also be used by those who cannot tolerate other treatments, including the elderly and frail.
This has the potential to provide a significant advantage over existing treatment options as it allows for a greater number of patients to be treated, including those who wish to be treated closer to home.
Earlier this month inobrodib was given Fast Track designation by the FDA.