Self-proclaimed pioneer of RNA-targeted medicine Ionis Pharmaceuticals says it is looking to develop an “even better medicine” to Spinraza to treat spinal muscular atrophy (SMA). At its annual meeting of stockholders earlier this month, Ionis Pharmaceuticals spoke about the success of its flagship molecule, Spinraza (nusinersen), an oligonucleotide approved by the US Food and Drug Administration (FDA) in December 2016 for the treatment of SMA. “Spinraza is the standard-of-care for all forms of the disease SMA. It is a medicine…

Complex pathologies and small patient populations make rare disease R&D difficult according to Alexion, which says recent FDA guidelines are welcome. At large biopharma firms, rare disease drug R&D projects are becoming common. Their high revenue potential combined with IP advantages afforded to such products makes them an attractive focus for developers. For example, orphan drug designation entitles firms to reduced fees, tax breaks for trials and extended market exclusivity. Investors also welcome such designation. According to one study, developers…

There is no one-size-fits-all model for cell and gene therapy pricing plans says Orchard Therapeutics, but industry must adapt a system set up for chronic care to incorporate curative one-off treatments. There have been questions over how payors and insurance firms would cope with such next-generation therapeutics have been asked ever since Novartis launched its first cell and gene therapy product Kymriah (tisagenlecleucel) in 2017 at a list price of $475,000. But the recent US Food and Drug Administration (FDA)…