Ionis: zilganersen PDUFA ditetapkan pada 22 September 2026

FDA telah menetapkan tanggal aksi PDUFA pada 22 September 2026 untuk zilganersen di bawah Tinjauan Prioritas. Siaran pers juga menyajikan data studi pivotal tambahan yang positif di seluruh titik akhir motorik, yang dilaporkan pasien, dan yang dilaporkan klinisi yang mendukung hasil topline yang telah diumumkan sebelumnya.

Fakta utama

Diajukan: 21 Apr 2026, 11.00
Peristiwa: Tanggal PDUFA ditetapkan
Arah: Positif
Perusahaan: IONSIonis
Aset: zilganersen
Sumber: SEC 8-K
Keyakinan: Bersumber
Kalender: Keputusan FDA (PDUFA) · 22 Sep 2026 · Positif
GFAP p: 0.003
10MWT p: 0.041
GMFM-88 p: 0.034
penurunan GFAP: 33.6%
perbedaan 10MWT: 33.3%
perbedaan GMFM-88: 22.9 poin

Kutipan sumber

EX-99.1 2 ef20071051_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 FINAL: April 20, 2026 Ionis presents new data from pivotal study of zilganersen in Alexander disease (AxD) at AAN 2026 Annual Meeting – New data highlight potential treatment benefit across multiple AxD symptom domains, reinforcing zilganersen’s positive impact on people living with this rare, often fatal neurological disease – – PDUFA date set for September 22, 2026 – CARLSBAD, Calif., April 21, 2026 -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. These findings, which will be prese

SEC 8-K