PIM designation in the UK for GBT's voxelotor
US biopharma Global Blood Therapeutics (Nasdaq: GBT) says that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted a Promising Innovative Medicine (PIM) designation for voxelotor for the potential treatment of hemolytic anemia in adults and adolescent patients 12 years of age and older with sickle cell disease (SCD).
Following a review by the MHRA, PIM designations are given to promising treatments that are likely to offer a major advantage for patients and are an early indication that the treatment is a promising candidate for the Early Access to Medicines Scheme (EAMS). For the MHRA to grant a PIM designation, the product must meet each of the following three criteria:
- The condition should be life-threatening or seriously debilitating with high unmet need, meaning there is no method of treatment, diagnosis or prevention available, or existing methods have serious limitations.
- The medicinal product is likely to offer major advantage over methods currently used in the UK, based on both non-clinical and clinical data.
- The potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit risk balance.
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