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Latest From Lucie Ellis
Orphan drug development used to be considered a license to print money, with high price tags and a focused customer base. With the development of an increasingly tough orphan drug pricing environment in the US and Europe, biotechs are adopting new strategies. CNS drug developer Minoryx will introduce endpoints into its Phase II/III studies for rare brain disease X-ALD that should help demonstrate both efficacy and value for future pricing discussions.
The microbiome market will see its value rise to $500m by 2022, and higher during the next decade. The key driver will be the increase in biotech and pharma firms looking at the emerging research space for new therapeutic options to treat chronic conditions such as Crohn’s disease.
New NICE and NHS England appraisal rules regarding timelines and price thresholds could fast-track new cost-effective drugs but then delay them from reaching patients for up to three years. The new regulations, which will come into force in April, have raised a lot of questions around methodology and impact outside of England.
New NICE and NHS England appraisal rules regarding timelines and price thresholds – labeled stark and contradictory – could fast-track new cost-effective drugs but then subject the same products to significant delays of multiple years.
Editas' stock shot up 11% by mid-morning on March 14, after the gene therapy company announced a deal with Allergan worth $90m up front for "cool" CRISPR programs in ocular indications. However, the lead therapy included in the deal offers potentially limited profitability in a small orphan space.
North Carolina biotech may need to conduct a 9,000-patient safety study after solithromycin was rejected by FDA, but it has $231m in cash and another Phase III candidate. Apricus is clearing financial room by transferring ex-US rights to Vivaros to Ferring, while Heat is acquiring former spinout Pelican.