Part 1: Execs from rare disease-focused companies weighed in on investor sentiment during a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference.
With the help of regulatory incentives for orphan drugs, including additional exclusivity and priority review vouchers, drugs for rare diseases have become hot commodities.
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The company’s FcRn inhibitor nipocalimab was approved by the US FDA for generalized myasthenia gravis (gMG), where it will join a competitive market.
The companies will co-develop the drug for additional indications beyond Huntington’s disease, while Prilenia retains rights in key global markets.
The German group is paying $3.9bn to get hold of the US firm and its two approved products.
After dropping its lead program in December, Spruce has been exploring strategic options – and thinks it has a de-risked path for MPS IIIB therapy tralesinidase from BioMarin.
BridgeBio reported $36.7m in Q1 2025 sales of its ATTR-CM drug Attruby, tripling consensus forecasts. Vyndaquel-maker Pfizer acknowledged the product’s impact during the quarter.
The San Diego biotech’s lead product candidate is an antisense oligonucleotide-based therapy slated to enter Phase III development in the third quarter.
The company’s FcRn inhibitor nipocalimab was approved by the US FDA for generalized myasthenia gravis (gMG), where it will join a competitive market.
