List of signals discussed at PRAC since September 2012

PRAC recommendations on signals adopted at the 10-13 March 2025 meeting

Medicine shortage communications (MSC)

Medicine shortage communication (MSC): Mimpara (cinacalcet)

Mimpara

Orphan designation: single guide RNA containing a sequence complementary to human ALB locus gene, intron 1, target region, Ziclumeran Treatment of haemophilia B, 25/03/2025 Positive

Orphan designation: povetacicept Treatment of primary IgA nephropathy, 25/03/2025 Positive

Orphan designation: extract from Cannabis flower, containing high levels of cannabidiolic acid and <0.3% of tetrahydrocannabinol, Extraction solvent: olive oil, virgin Treatment of Rett syndrome, 25/03/2025 Positive

Orphan designation: allopurinol Treatment of Marfan syndrome, 25/03/2025 Positive

Orphan designation: adeno-associated virus vector serotype 8 containing the human F9 gene Treatment of haemophilia B, 25/03/2025 Positive