rare diseases

Latest Headlines

Latest Headlines

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.

AstraZeneca snags $300M from Sanofi for rare cancer med amid cash-generating deal spree

AstraZeneca CEO Pascal Soriot has long argued that shedding some of the company's noncore businesses could help jump-start sales, adding cash to its reservoirs as it chases its lofty sales goals. Now, the British drugmaker is taking another step in that direction as it passes off one of its rare cancer drugs to Sanofi, getting $300 million in the process.

BioMarin shines as dwarfism drug boosts growth in children

Three years ago, BioMarin top scientist Henry Fuchs pitched BMN-111 as one of the company's most promising early-stage drug prospects, with stellar mouse data to suggest that it was on track to develop a therapy to correct the genetic mutation behind dwarfism. On Wednesday evening, the San Rafael, CA-based biotech outlined Phase II data from 26 children that provided ample proof-of-concept human data that Fuchs was well justified in his belief.

Sage gets a peek at success treating postpartum depression, and its shares soar

Developing new drugs for depression has become one of the most frustrating tasks in biotech as companies have struggled to demonstrate a positive effect against hard-to-control placebo responses in clinical trials or prove that dramatic and quick responses in small groups can translate into safe and broadly used drugs. Sage Therapeutics, though, is touting the results it's seen in just four patients.