First Promising Innovative Medicine designation for self-administered treatment of rare disease
pharmafile | February 17, 2022 | News story | Business Services |
UCB has announced that zilucoplan, its developmental treatment for adults with generalized myasthenia gravis (gMG) has been granted Promising Innovative Medicine (PIM) designation in the UK by the MHRA. This marks the first investigational treatment with a published PIM designation for gMG in the UK.
Zilucoplan is a self-administered, subcutaneous (SC) peptide inhibitor of complement component 5 (C5).
“Myasthenia gravis is a serious condition that can have unpredictable and debilitating symptoms, affecting patients’ lives in multiple ways,” Claire Brading, Managing Director and Head of Neurology UCB UK & Ireland commented. “We are pleased that zilucoplan has been recognised by the MHRA for its potential to address the urgent need for treatment innovation in this area. This is testament to UCB’s commitment to MG patients in the UK, as well as to harnessing science to bring benefit to people living with rare diseases more widely.”
The granting of a Promising Innovative Medicine designation is an early indication that an investigational treatment may be eligible for an EAMS licence, which can provide patients with life threatening or seriously debilitating conditions pre-approval access to medicines where there is a clear unmet medical need. PIM is only granted to products that treat life-threatening or seriously debilitating conditions, with special emphasis on patient quality of life, and where there is high unmet need due to no available method treatment, or serious limitations to existing methods.
PIM designation is also only granted to medicinal products likely to offer a major advantage over current methods in the UK, and where the benefits of the product are likely to outweigh adverse events.
Zilucoplan has been awarded the designation for gMG, which is a chronic auto-immune disease that disrupts the way the nerves communicate with the muscles. MG is a rare disease, with an estimated 12,000 people in the UK living with the condition. People living with MG can experience a variety of symptoms, including drooping eyelids, double vision and difficulty swallowing, chewing and talking, as well as severe muscular weakness that can result in life threatening weakness of the muscles of respiration.
Ana Ovey
