PERTH, Australia – With the launch of its lead product, Scenesse (afamelanotide), in the U.S. and Europe, Melbourne-based Clinuvel Pharmaceuticals Ltd. is expanding its global footprint and widening its pipeline as it opens a new R&D center in Singapore and builds up its franchise in photo medicine.
The first weekly therapy for adult growth hormone deficiency (GHD) has arrived in the U.S. with FDA approval for Novo Nordisk A/S' Sogroya (somapacitan). It’s the first of Novo's regulatory filings for the medicine, also submitted in the EU and Japan, to win approval. Sogroya replaces endogenous growth hormone in adults who don't produce enough of it. Novo said it is working to finalize plans to make the therapy available and has not yet disclosed pricing.
Tricida Inc. executives were not surprised when the FDA issued a complete response letter for veverimer to treat chronic metabolic acidosis. Despite an Aug. 22 PDUFA date, the company had received an FDA notification on July 16 that there were deficiencies that would preclude discussion of labeling and postmarketing requirements and commitments.
As Carmel, Ind.-based MBX Biosciences Inc., taking aim at rare endocrine diseases, disclosed $34.6 million in a series A financing, CEO and co-founder Kent Hawryluk told BioWorld that “everyone on the leadership team we’ve worked with in prior lives, so to speak,” adding that he’s “excited to have the band back together.”
A month ahead of its PDUFA date, Ultragenyx Pharmaceutical Inc.’s NDA for triheptanoin (UX-007) won FDA approval. Branded Dojolvi, the highly purified, synthetic, seven-carbon fatty acid triglyceride was cleared for use in treating pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
DUBLIN – Gene therapy developer Freeline Therapeutics Ltd. added $80 million in new investment to take its series C round to $120 million in total. The new cash will help to fund a pivotal trial of its lead gene therapy program in hemophilia B, enable it to continue a phase I/II trial of a gene therapy in Fabry disease and allow it to progress its preclinical programs in Gaucher disease and hemophilia A, while also making ongoing investments in its adeno-associated virus (AAV) gene therapy technology and its manufacturing platform.
San Diego-based Autobahn Therapeutics Inc.’s $76 million series B round will let the firm advance lead candidate ABX-002, a thyroid hormone receptor beta agonist therapy for multiple sclerosis and adrenomyeloneuropathy, a rare genetic disorder, plus a portfolio of central nervous system programs that leverage the company’s brain-targeting chemistry platform.
DUBLIN – Alnylam Pharmaceuticals Inc. is on track to secure its third FDA approval in successive years, as its siRNA drug, lumasiran, hit all its marks in a phase III trial in patients with primary hyperoxaluria type 1 (PH1). The drug is already undergoing regulatory review and has a Dec. 3 PDUFA action date. It is undergoing accelerated assessment in Europe as well.
The $65 million that Hotspot Therapeutics Inc., of Boston, raised will help advance its lead programs to the clinic, including protein kinase C antagonists for Th2 and T-reg-driven autoimmune disease and S6 kinase antagonists for rare metabolic disease.
Kriya Therapeutics Inc., a startup led by former Axovant Sciences Inc. executive Shankar Ramaswamy, has raised $80 million in series A financing expected to help advance a portfolio of gene therapies for diabetes and other chronic conditions.
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