The FDA approved the combination of elexacaftor, ivacaftor, and tezacaftor (Trikafta, Vertex Pharmaceuticals) on October 21 for the treatment of patients age 12 years or older with cystic fibrosis (CF) and at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. The combination was approved almost 6 months early (3/19/2020 PDUFA date).
Ascendis plans to file a BLA for TransCon hGH in the first half of 2020 and an MAA in in the second half of 2020 to treat pediatric growth hormone deficiency. TransCon hGH was designated an Orphan drug by the EMA.
The EMA has granted teplizumab PRIME status for the prevention or delay of clinical type 1 diabetes
Announced Research Updates
Reata announced that in the 48-week, 103 patient Phase II, MOXIe trial, patients treated with omaveloxoloneimproved the modified Friedreich ataxia Rating Scale by 1.55 points compared to a worsening of 0.85 points with placebo in patients with Friedreich ataxia.
In March Biogen announced that an interim analysis of the Phase III ENGAGE and EMERGE trials found a low likelihood that aducanumab would improve or slow cognition in Alzheimer’s patients, so Biogen discontinued both trials in March 2019. Biogen analyzed additional data from 3,285 patients and found an improvement in 547 patients enrolled in EMERGE that received the highest dose of the drug. Aducanumab reduced the decline in CDR-SB (23%), MMSE (15%), ADAS-Cog 13 (27%) and ADCS-ADL-MCI (40%). The expanded cohort of high dose patients in ENGAGE did not demonstrate an improvement compared to placebo.
Novartis announced that in two 12-week, 700 patient Phase III trials (ZEAL 1 and ZEAL 2), treatment with fevipiprant did not improve FEV1 compared to placebo in patients with moderate asthma. Fevipiprant is being evaluated in preventing exacerbation in moderate to severe asthma in the Phase III LUSTER 1 and 2 trials.
Protalix announced interim results from 16 patients in the 12-month, 22 patient, Phase III, open-label BRIDGE trial, showed a decline in eGFR from -5.10 mL/min/1.73m2 per year on stable doses of agalsidase alfa to -0.23 mL/min/1.73m2 per year on pegunigalsidase. Protalix plans to submit a BLA for pegunigalsidase in 1Q20.
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