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Pipeline News and Updates
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Update for June 22, 2022

6/22/2022

 
Regulatory Update
 
The FDA approved vutrisiran (Amvuttra, Alnylam), on 6/13/2022, for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Alnylam set WAC for vutrisiran at $463,500 per year.
 
The FDA extended the PDUFA date for omecamtiv mecarbi by three months to allow additional time to review newly submitted pharmacokinetic data. The new PDUFA date is 2/28/2023.
 
Mallinckrodt resubmitted an NDA for terlipressin for the treatment of hepatorenal syndrome - acute kidney injury (HRS-AKI).
 
Addex Therapeutics discontinued development of dipraglurant.
 
The FDA designated rencofilstat an Orphan Drug for treatment of hepatocellular carcinoma.
 
Canada approved the combination of sodium phenylbutyrate and taurursodiol (AMX0035) for the treatment of Amyotrophic Lateral Sclerosis (ALS).
 
Announced Research Updates
 
BridgeBio announced that in the 24-week period 3 of a Phase IIb trial, continued treatment with encaleret resulted in parathyroid hormone levels of 31.3 pg/ml and urine calcium excretion or 189 mg/24 hour in patients with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
 
Roche announced that in a 260 to 416 week, 252 patient, Phase II trial, treatment with crenezumab did not slow the decline in the Alzheimer's Prevention Initiative Autosomal-Dominant Alzheimer's Disease (API ADAD) Composite Cognitive Test Total Score and Episodic Memory Measure: Free and Cued Selective Reminding Task (FCSRT) score in cognitively unimpaired individuals who have no clinical symptoms of Alzheimer’s disease and carry the PSEN1 E280A autosomal dominant mutation.
 
Published Research Updates
 
In a draft report, ICER found evidence to support efficacy for AMX0035 in the treatment of amyotrophic lateral sclerosis (ALS) to be comparable or better compared to standard of care alone. If AMX0035 is priced similar to intravenous edaravone at $171,000 a year, the drug would exceed cost effectiveness thresholds.


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