Rare Disease

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AZ preps FDA filing for $3.6bn amyloidosis drug eplontersen

Armed with new phase 3 data, AstraZeneca is preparing to file for regulatory approval of eplontersen, an antisense drug for one of the complications of the disease transthyretin amyloidosis

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Genetic engineeering concept with 3d rendering robotic hand editing dna helix

Second unanimous FDA adcomm vote boosts bluebird bio

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern.

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FDA panel backs bluebird's CALD gene therapy, despite safety...

The FDA may have safety concerns abut bluebird bio's gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks.

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FDA grants speedy review to Dupixent rare skin disease

Sanofi and Regeneron should hear from the FDA in the autumn whether it will approve their blockbuster immunology drug Dupixent as a therapy for rare skin disease prurigo nodularis (PN).

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UK grants early access to Argenx' efgartigimod for myastheni...

The UK regulatory authority has cleared the path for some patients with generalised myasthenia gravis (gMG) to get early access to Argenx' efgartigimod while it reviews the marketing applic

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PTC gets green light for gene therapy Upstaza in EU

PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f