Scrip

Alnylam Seeking Lumasiran Approvals Soon, But Has Two Rivals On Its Heels

By Mandy Jackson 27 Nov 2020

Phase III results support early 2020 filings for what could be the RNAi specialist’s third commercial product, but two competing drugs for primary hyperoxaluria from Dicerna and OxThera aren’t far behind. 

Topic Clinical Trials Launches Rare Diseases

Pink Sheet

US FDA's Regulatory Flexibility To Be Tested As Delcath Overhauls Melphalan Trial Design

By Sue Sutter 22 Aug 2018

Accrual difficulties force changes that nullify Special Protocol Assessment; ongoing, randomized FOCUS trial in patients with ocular melanoma that has metastasized to liver will become single-arm study and target enrollment will be reduced by 66%.

Topic Clinical Trials Approvals Rare Diseases FDA

Pink Sheet

Pharma Wants Clarity On Joint Evaluation Of EU Paediatric And Orphan Regulations

By Vibha Sharma 19 Jan 2018

The European Commission's plans to evaluate the EU pediatric and orphan drugs regulation in tandem.

Topic Rare Diseases

Topic Biosimilars Rare Diseases

Scrip

Duchenne Muscular Dystrophy: The Race For The $1bn Opportunity

By Lucie Ellis 03 Oct 2017

With three products now approved in Europe and/or the US for Duchenne Muscular Dystrophy

Topic Rare Diseases

Pink Sheet

Orphan Biosimilar: Soliris Is One Target

13 Jun 2017

Using lessons learned from the first wave of biosimilar approvals – and citing high prices in the US – some developers are starting to 'think small' with biosimilars for rare disease markets. Alexion’s Soliris is a case in point.

Topic Biosimilars Rare Diseases Research & Development

Pink Sheet

Disease Awareness Campaigns Still Must Tread Carefully, Observer Says

By Mark W. Sherman 03 Jun 2017

Sponsored plotline for General Hospital was careful to avoid irking US FDA; agency withdrew its guidance on disease awareness campaigns in 2015.

Topic Rare Diseases

Pink Sheet

BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs

By Emily Hayes 03 May 2017

Filing supporting Brineura was supported by a tiny, single-arm study and had no advisory committee review, the latest sign of the US regulatory agency's willingness to speed orphan drugs for serious pediatric diseases.

Topic Rare Diseases