The first therapies for several rare diseases were among medicines given the green light by European regulators at their monthly meeting. The EMA’s Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion for Sanofi SA’s Xenpozyme (olipudase alfa) for two types of Niemann-Pick disease and Eiger Biopharmaceuticals Inc.’s Zokinvy (lonafarnib) for children with Hutchinson-Gilford progeria syndrome or progeroid laminopathies. PTC Therapeutics Inc.’s Upstaza (eladocagene exuparvovec), the first medicine for adults and children with aromatic L-amino decarboxylase deficiency, was also backed by the CHMP.
Keros Therapeutics Inc. announced preliminary results from a phase I trial of its engineered ligand trap KER-012 that gave its team confidence to proceed with larger studies in pulmonary arterial hypertension (PAH) and potentially some bone diseases. But company shares (NASDAQ:KROS) fell 16.6% to $38.50 May 18, following the announcement, perhaps over concerns about trial subjects that emerged in a company-hosted investor call.
It was a patient-reported outcome, one that could actually be seen in the mirror, that alerted researchers they might be on track in their phase I/II study of cystinosis. The patient noticed that for the first time in his life his hair had become darker, like his brother’s. It was all because the rare disease inhibiting the pigment in his body was being impacted by the treatment. “It’s a secondary issue, but I find it fascinating,” Avrobio Inc.’s CEO, Geoff MacKay, told BioWorld. “When you run trials like this, you stumble upon some fascinating results.”
Pacific Biosciences of California Inc.’s technology could rapidly increase the number of rare diseases—and their causes—identified by sequencing. Researchers at Children’s Mercy Research Institute in Kansas City found four times as many rare coding structural variants using Pacbio’s highly accurate long reads (Hifi) sequencing than standard sequencing detected. Results of the study were published in Genetics in Medicine.
Alexion Pharmaceuticals Inc. has demonstrated the commercial potential for rare disease drugs with its complement inhibitor Soliris (eculizumab) and long-lasting follow-up Ultomiris (ravulizumab) driving blockbuster sales. A host of other companies are hoping to compete with Ultomiris, which is U.S. FDA-approved for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Shares in Pharming Group NV were up after the company announced detailed data from its phase II/III trial of leniolisib for the rare disease activated phosphoinositide 3-kinase delta (PI3K-delta) syndrome (APDS).
As it settles a patent dispute with Japan’s Chugai Pharmaceutical Co. for $775 million, the U.K.’s Astrazeneca plc is looking to bring intellectual property in-house by snapping up a new generation of talented scientists.
As it settles a patent dispute with Japan’s Chugai Pharmaceutical Co. for $775 million, the U.K.’s Astrazeneca plc is looking to bring intellectual property in-house by snapping up a new generation of talented scientists.
Biotechs that tackle the effects of aging are beginning to make headlines: in January Altos Labs Inc. launched with a reported investment from Jeff Bezos. With Bezos getting involved with San Francisco-based Altos, the immediate reaction was that anti-aging biotechs would be there for the benefit of billionaires searching for eternal life.
Not so, according to London U.K.-based Genflow Biosciences plc, which hopes to show that fighting aging is really about improving health as people age.
Novo Nordisk A/S, best known for its range of diabetes drugs and insulins, has said that growing its rare disease unit will be an important part of its business strategy this decade. Novo Nordisk has actually been operating in rare diseases for 40 years, starting in hemophilia and gradually growing its presence. But the Bagsværd, Denmark-based company has big plans for its rare diseases arm in the next few years, with a pipeline of drugs aimed at the busy hemophilia market and for rare endocrine disorders.
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