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PTC’s ‘Life-Changing’ Gene Therapy Among Nine Drugs To Win EU Thumbs Up

 
• By Neena Brizmohun

The European Medicines Agency has delivered its verdict on the latest medicines it believes should be approved for marketing in the EU. By contrast, it has given the thumbs down to one biosimilar anticancer drug and its duplicate.

A gene therapy for a rare nervous system disorder could be approved soon • Source: Alamy

PTC Therapeutics’ Upstaza (eladocagene exuparvovec) has stepped closer to becoming the first ever treatment approved for the rare and fatal nervous system disorder, AADC deficiency, after the European Medicines Agency recommended that the one-time gene replacement therapy should be granted pan-EU marketing authorization.

Once the EMA’s recommendation is ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for AADC (aromatic L-amino acid decarboxylase) deficiency for patients 18 months and older

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More from Europe

EU Pharma Reform: Exclusivity Vouchers Alone Are Insufficient Incentive For Antimicrobials, Industry Says

 
• By Eliza Slawther

European pharma trade associations EFPIA and EUCOPE outline their respective views on how the EU’s pharma legislation overhaul should tackle antimicrobial resistance, and why transferable exclusivity vouchers alone will not suffice as incentives.

What The EMA Can Teach HTA Bodies About Joint Clinical Assessments

 
• By Francesca Bruce

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

Switzerland Simplifies Imports Of Unauthorized Drugs To Tackle Drug Shortages

 
• By Eliza Slawther

Swiss authorities have introduced temporary measures that will make it easier for health care professionals to import medicines that are either not authorized or not available in Switzerland, which will particularly benefit pediatric drugs, in light of ongoing shortages.

EU ‘Can’t Expect Industry To Give Up Proprietary Insights’ Under Health Data & Clinical Trials Regulations

 
• By Eliza Slawther

Both the EU Clinical Trials Regulation and the European Health Data Space Regulation have the potential to improve harmonization and be highly valuable for industry – but the importance of protecting company data will be paramount, a life sciences consultant says.

More from Geography

Pharma Predicts Modest Tariff Impact, Depending On What Comes Next

 
• By Jessica Merrill

Drugmakers are not expecting a big financial hit from tariffs for now, but a report commissioned by the industry trade association PhRMA suggests a potentially steep cost for pharma-sector tariffs.

What The EMA Can Teach HTA Bodies About Joint Clinical Assessments

 
• By Francesca Bruce

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

Moderna Pivots To Increase Focus On Cancer Amid US Vaccine Policy Changes

 
• By Joseph Haas

During its first quarter earnings call, Moderna avoided criticizing vaccine policy changes, but de-emphasized its flu/COVID-19 vaccine for those under age 50 and prioritized cancer programs.