Tag: Rare Disease
PharmaSignal — Rare Disease therapeutic area
Ionis’ antisense drug stabilizes ultra-rare disease ahead of FDA decision
BioSpace
The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.
Italy leads the way on child genetic disease diagnosis
Pharmaphorum
A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.
221: An Interview With Dutch Neurologist Ewout Groen of SMA Europe
Rare Disease Audio
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dutch neurologist Ewout Groen, PhD, a member of the Scientific Advisory Board of SMA Europe.
Travere wins long-awaited approval for kidney disease drug
BioPharma Dive
The clearance of Filspari in a condition known as FSGS ends a winding regulatory journey and unlocks a revenue opportunity believed to be worth more than $1 billion annually.
Travere Therapeutics gains FDA approval for sparsentan in FSGS
Pharmaceutical Business Review
The approval makes Filspari the first and only FDA-approved medicine to treat FSGS, expanding its use beyond IgA nephropathy (IgAN) into a second rare kidney condition.
Travere Therapeutics gains FDA approval for sparsentan in FSGS
Pharmaceutical Technology
Travere Therapeutics has received FDA approval for Filspari to reduce proteinuria in patients aged eight years and over with FSGS without nephrotic syndrome.
Travere claims first FDA OK in rare kidney disease FSGS
Pharmaphorum
Shrugging off an earlier phase 3 disappointment, Travere’s Filspari has become the first FDA-approved treatment for rare kidney disease FSGS.
FDA seeks permanent future for rare pediatric priority review vouchers
Pharmaceutical Technology
The rare disease framework has had a turbulent past few years, with the FDA looking to create regulatory stability.
Neurocrine to acquire Prader-Will drug in $2.9B Soleno buyout
BioPharma Dive
The deal hands Neurocrine a controversial medicine, Vykat, that achieved $190 million in sales last year but faces persistent questions about its growth trajectory and safety, according to Wall Street
Neurocrine Nabs Rare Obesity Disorder Drug in ‘Surprising’ $2.9B Soleno Deal
BioSpace
While an acquisition is a good exit for Soleno Therapeutics, the company’s acceptance of Neurocrine Biosciences’ $53-per-share offer came as a surprise to Stifel analysts given the potential growth of
Rare Disease Leaders Call for Regulatory Clarity as FDA Balances Urgency With Rigor
BioSpace
With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore re
FDA approves new high dose Spinraza regimen for SMA
Biopharma M&A Heats Up, Rare Diseases Win Three Approvals, Wave Crashes
BioSpace
Biogen, Eli Lilly, Merck and Novartis spent more than $20 billion to absorb biotechs with promising or approved drugs; the rare disease space notched approvals for therapies from Denali Therapeutics,
Biogen uplifts rare disease pipeline with $5.6bn Apellis buyout
Pharmaceutical Technology
Biogen’s acquisition of Apellis Pharmaceuticals will add Syfovre and Empaveli to the latter company’s portfolio of medicines.
PepGen Loses Half of Share Price on Mixed Data in Rare Muscle-Wasting Disease
BioSpace
PepGen’s lead candidate for myotonic dystrophy type 1 barely beat the placebo in a Phase 2 trial in terms of fixing incorrect gene splicing, but the biotech attributed the poor result to an outlier.
UCB brings first therapy for rare disease TK2d to EU
Pharmaphorum
The EU has approved UCB’s Kygevvi for rare disease TK2d, and Sanofi’s acute GvHD therapy Rezurock, in a pair of decisions announced this morning.
AZ considers filings for long-acting Strensiq follow-up
Pharmaphorum
AstraZeneca’s long-acting Strensiq follow-up efzimfotase alfa has hit the mark in children with rare disease HPP, but the data in adults is unclear.
Biogen Bounces Back With FDA Nod for High-Dose Spinal Muscular Atrophy Drug
BioSpace
The FDA rejected the high-dose regimen of Spinraza in September last year due to manufacturing concerns.
The Rare Journey Needs a Special Pharmacy Partner
Drug Channels
Today’s guest post is from Bansi Nagji, Chief Executive Officer at PANTHERx Rare.
FDA clears Denali drug in ‘clear step’ for rare disease biotechs
BioPharma Dive
The approval of Denali s Hunter syndrome treatment, Avlayah, comes after a series of drug rejections and delays that had led to criticism of the FDA s stance on rare disease therapies.