Pharming gets promising innovative medicine tag in UK for leniolisib for rare disorder

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Pharming Group (NASDAQ: PHAR) said the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted promising innovative medicine (PIM) designation to leniolisib to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a rare immunodeficiency disease.

The company added that it also received a positive decision from the MHRA on a Pediatric Investigation Plan (PIP) submission for leniolisib to treat APDS in patients ages 1-year to less than 18 years of age.

Pharming (PHAR) noted that a PIP is a development plan aimed at ensuring that the necessary data is obtained to support the marketing authorization of a medicine for use in children.

The company said leniolisib PIP includes two planned global trials, the first in children ages 4-11 years and the second in children ages 1-6 years with APDS.

The company expects to begin recruitment for the program in H2 2022.

Pharming (PHAR) plans to begin submitting global registration filings for leniolisib in Q2 2022 and, if approved, intends to launch therapy in the US in Q1 2023 and starting a series of European launches in H2 2023.

APDS is a rare immunodeficiency disease that affects 1 to 2 people per million and is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells, the company said in an April 26 press release.

gorodenkoff / iStock via Getty Images Pharming Group (NASDAQ: PHAR) said the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted promising innovative medicine (PIM) designation to leniolisib to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a rare immunodeficiency disease. The company added that it also received a positive decision from the MHRA on a…

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