Alnylam shares take a hit amid sluggish sales of first drug
Shares of Alnylam Pharmaceuticals fell Wednesday after the Cambridge biotech reported a slow uptake of its first FDA-approved drug, an expensive, first-of-its-kind treatment for a rare disease that is difficult to diagnose.
In August, Alnylam (Nasdaq: ALNY) became the first company to receive FDA approval for a drug using a new gene-silencing technology called RNA interference, or RNAi. The drug, Onpattro, treats a rare disease called hereditary ATTR amyloidosis that is hard to diagnose, which poses… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - November 7, 2018 Category: American Health Authors: Allison DeAngelis Source Type: news
New, more accessible staging system developed to predict survival for patients with AL Amyloidosis
(Boston Medical Center) A new staging system developed with a more accessible test to predict the chance of survival in patients living with light chain (AL) amyloidosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 7, 2018 Category: International Medicine & Public Health Source Type: news
FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy
Inotersen treatment led to improvements in neurologic and quality-of-life endpoints in patients with hereditary transthyretin amyloidosis with polyneuropathy in a pivotal phase 3 trial.FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - October 8, 2018 Category: Consumer Health News Tags: Rheumatology News Alert Source Type: news
Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. has turned the tide, receiving approval for another treatment for a rare genetic disease.
The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build up… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 8, 2018 Category: Biotechnology Authors: Allison DeAngelis Source Type: news
Cambridge's Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease.
The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - October 6, 2018 Category: American Health Authors: Allison DeAngelis Source Type: news
Cambridge's Akcea rebounds with FDA approval of first drug
After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease.
The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 6, 2018 Category: Biotechnology Authors: Allison DeAngelis Source Type: news
FDA Approves Tegsedi (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
BOSTON and CARLSBAD, Calif., Oct. 05, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the U.S. Food and Drug... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - October 5, 2018 Category: Drugs & Pharmacology Source Type: news
What Is to Blame for Cognitive Decline in Ageing? What Is to Blame for Cognitive Decline in Ageing?
What are the key drivers of cognitive decline in the elderly? A new study suggests that amyloidosis and neurodegeneration have both independent and synergistic effects.Brain (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - September 17, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news
What you missed on your summer vacation in Mass. biotech news
Here's a look at the Boston biotech news you missed during your summer vacation:
Alnylam gets historic FDA approval for first drug
Cambridge's Alnylam Pharmaceuticals (Nasdaq: ALNY) received FDA approval for its first drug on Aug. 10, a first-ever treatment for a rare genetic disease that uses RNA interference to silence genes. The drug treats hereditary ATTR amyloidosis, w hich causes the liver to protein abnormal proteins that impede nervous system and organ functions. Marketed under the name… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 4, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news
Periodic Fevers: What to Watch For
(MedPage Today) -- Complications include amyloidosis and end-stage renal disease (Source: MedPage Today Pediatrics)
Source: MedPage Today Pediatrics - August 28, 2018 Category: Pediatrics Source Type: news
ESC: Tafamidis Cuts Deaths, Admissions in TTR Cardiac Amyloidosis
(MedPage Today) -- Plus clear advantage seen in 6-minute walk test (Source: MedPage Today Nephrology)
Source: MedPage Today Nephrology - August 27, 2018 Category: Urology & Nephrology Source Type: news
Alnylam gets FDA nod for first-ever RNAi drug
The U.S. FDA has approved Alnylam Pharmaceuticals ’ Onpattro, making it the first medicine specifically cleared to treat a rare and deadly disease called hereditary transthyretin amyloidosis. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - August 13, 2018 Category: Pharmaceuticals Source Type: news
FDA Approves Onpattro (patisiran) Targeted RNA-based Therapy for Polyneuropathy Caused by hATTR
August 10, 2018 -- The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - August 10, 2018 Category: Drugs & Pharmacology Source Type: news
Patisiran Wins Two Firsts With FDA Approval
(MedPage Today) -- RNAi drug OK ' d to treat polyneuropathy from hereditary transthyretin amyloidosis (Source: MedPage Today Primary Care)
Source: MedPage Today Primary Care - August 10, 2018 Category: Primary Care Source Type: news
Cambridge biotech Alnylam wins FDA approval for first drug
The FDA has approved Alnylam Pharmaceuticals Inc.'s first drug, a novel rare disease treatment that analysts believe could eventually top $1 billion in annual sales, marking a major milestone for one of the state's fastest-growing biotechs.
The FDA said Friday it had approved Cambridge-based Alnylam's (Nasdaq: ALNY) drug patisiran, which targets a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. The disorder, which affects around 50,000 people worldwide,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 10, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news
