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Hereditary Angioedema Patients Urge Work On Dosing, Product Shortages

 
• By Kate Rawson

US FDA's final patient-focused drug development meeting shows high interest clinical trial enrollment.

Hereditary angioedema patients used the final US FDA-hosted patient-focused drug development meeting to advocate for the development of additional treatment options for HAE, citing intermittent manufacturing supply issues with existing biologics and the lack of a treatment that sufficiently prevents or controls body swelling episodes.

HAE is a rare genetic disorder that is associated with painful episodic, recurrent attacks of body swelling caused by abnormalities in the C1-esterase inhibitor protein

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EU HTA Regulation: ‘Stepping Beyond’ JCAs May Be Needed For Very Rare Disease Drugs

 
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Cooperation between health technology assessment bodies across the EU will lead to a better joint clinical assessment process over time, but patients cannot afford a lengthy wait for improvements, speakers at a cell & gene therapy conference said.

US FDA Not Industry’s ‘Sock Puppet,’ Ultragenyx’s Emil Kakkis Says

 
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Vertex’s Alyftrek For CF Among Six Orphans On Track For Pan-EU Approval

 
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The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.

EMA To Consider Approvals For Eight Orphans, Including Vertex’s CF Drug Alyftrek

 
• By Francesca Bruce

The European Medicines Agency is this week expected issue opinions on whether the European Commission should grant EU marketing authorization for 19 new products.

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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.