rare diseases

Latest Headlines

Latest Headlines

Shire is paying $5.9B for Dyax in a defensive rare disease deal

Shire signed a deal to buy Dyax for $5.9 billion, planning to snatch up a smaller competitor and fortify its rare disease business without taking its eyes off of acquisition target Baxalta.

Marquee VCs back a $40M round for Crinetics' acromegaly drug

A group of marquee venture investors has joined forces to back a $40 million Series A for Crinetics Pharmaceuticals, an early-stage biotech in San Diego that has been working on a next-gen oral treatment for acromegaly.

Apellis swings for an $86M IPO amid a dicey climate for biotech

Apellis Pharmaceuticals, at work on treatments for rare diseases, is lining up to go public in an $86 million IPO as biotech's long-running hot streak looks all the more tenuous.

On second thought, Amicus won't be filing its lead drug this year

Amicus Therapeutics, a biotech comeback story, is walking back plans to submit its rare disease treatment for FDA approval this year, rethinking its whole regulatory strategy after follow-up conversations with the agency.

Amicus plots an end-of-year FDA filing for its resuscitated lead drug

Amicus Therapeutics, fresh off reviving a once-failed drug, has reached an accord with U.S. regulators and is on track to submit its rare disease treatment for FDA approval before the end of this year.

Regeneron team IDs a candidate for ultra-rare bone disease

Back at the beginning of 2014, a fast-growing Regeneron revealed that it had been building a new genetics research center that would zero in on carefully targeted diseases as it built new drug development programs from the ground up. Eighteen months later the big biotech says the effort--led by some top researchers wooed in to run the operation--has paid off with a clear target for a rare genetic bone disease. And they have an antibody in preclinical testing that could correct the disorder.

Cortendo swings for $86M IPO to fuel late-phase orphan drug R&D drive

Swedish-American biotech Cortendo is pitching to raise $86 million (€76 million) on Nasdaq to pump into a pair of late-phase trial programs, the more advanced of which could lead to a regulatory submission in the back half of 2017.

Alexion wins a pair of EU approvals to build out its rare disease ranks

Alexion Pharmaceuticals is no longer a one-product company, winning tandem European approvals for two rare disease drugs it hopes will mimic the success of Soliris, the world's most expensive treatment.

Amicus bags PhIII rare disease drug in $847M Scioderm buyout

Amicus Therapeutics has stepped in to snap up the late-stage rare disease biotech Scioderm, beefing up its orphan drug pipeline in exchange for $229 million in stock and cash along with a promise of up to $618 million more for meeting a slate of milestones.

Pfizer, OrbiMed back $40M Rhythm round for rare genetic disease R&D

Boston-based Rhythm has gone back to the venture well to draw up a fresh $40 million in backing from some A-list investors to fuel a mid-stage program for rare genetic disorders linked with a lethal level of obesity.