Global Markets

Making rare disease successes more common

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…

On the origin of serum: Controlling FBS supply, a riposte from the ESPA

Do not confuse the methods and purposes for controlling the serum supply chain by misleading end-users into believing that some origins of serum are better and safer than others, says the European Serum Products Association (ESPA). Last month, Bioprocess Insider published an article entitled: ‘Quality FBS, or just BS? Industry turning to supply chain certification’. The article detailed a presentation from the International Serum Industry Association (ISIA) at BPI Europe in Vienna, Austria and highlighted the application of independent audits,…

Personalized medicines need personalized pricing plans, Orchard

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)…

Will GSK have the capacity to bring Shingrix to China?

GlaxoSmithKline (GSK) has received Chinese approval for its shingles vaccine Shingrix, but with demand already outstripping supply the firm must invest significantly in its production capabilities. China’s National Medical Products Administration (NMPA) has approved Shingrix, a non-live, recombinant subunit vaccine for the prevention of shingles (herpes zoster) in adults aged over 50. Shingrix underwent an expedited review after falling on a list of 48 ‘clinically urgently needed new medicines’ in China aimed at bringing critical new prevention and treatment options…

Novartis wins approval for $2.1m gene therapy Zolgensma

Novartis says it is ready to launch Zolgensma (onasemnogene abeparvovec) within the next few weeks after receiving FDA approval for its spinal muscular atrophy (SMA) single-dose gene therapy. Zolgensma, a single-dose, one-time gene therapy has been approved to treat children less than two years of age with spinal muscular atrophy (SMA). “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” acting US Food and Drug Administration (FDA) commissioner…

WuXi scouts single-use site for Chinese vaccine joint venture

WuXi Biologics has formed a vaccine production division to service a $3 billion strategic partnership with an unnamed global pharma. WuXi Vaccines is a joint venture between contract development and manufacturing (CDMO) WuXi Biologics and Shanghai-based Hile Bio-Technology. The new entity has been formed to service a Letter of Intent (LOI) inked with a multinational vaccine company. The client has not been disclosed, but WuXi Biologics CEO Chris Chen told Bioprocess Insider the strategic partnership will allow the vaccine developer…

Pfizer urges FDA for flexibility in considering interchangeability studies

The US FDA has published long-awaited final guidance on the interchangeability of biologics, calling biosimilar developers to carry out switching studies. At the last count, there were 19 biosimilar products approved by the US Food and Drug Administration (FDA) US though only a handful have been launched. None of these products have been deemed interchangeable, meaning they cannot be substituted for the reference biologic at the pharmacy level without the involvement of a physician. However, this theoretically could change going…

Training and ‘sea turtles’ support WuXi Biologics’ growing China footprint

WuXi Biologics talks Chinese talent recruitment as it begins building an eighth biomanufacturing facility in the country, this one with 48,000 L of single-use bioreactor capacity. The latest addition to contract development and manufacturing organization (CDMO) WuXi Biologics’ bioproduction network is a 1.3 million square-foot center in Chengdu, Southwest China. The facility will include drug development and commercial manufacturing capabilities and boast 48,000 L in bioreactor capacity. According to CEO Chris Chen, the capacity breaks down to 12 x 4,000…

MilliporeSigma and Thermo contribute to Biolife’s ‘blowout’ Q1

Distributors STEMCELL Technologies and VWR also cited as sales of Biolife Solutions’s bio-preservation media and automated thawing products increased 51% year-on-year. For the first quarter 2019, Biolife, which makes and supplies biopreservation media and automated cell thawing media devices for cell and gene therapies, reported record revenues of $5.8 million (€5.2 million). “Dead cells don’t cure cancer, and the combined therapeutic, and economic risks our customers are facing should broaden use of our products as a best practice in the…

Subcutaneous success a boon for Halozyme, but biosimilars hit royalties

Roche’s subcutaneous Herceptin (trastuzumab) recently became the third product to be approved using Halozyme Therapeutics’s ENHANZE drug delivery tech. However, Halozyme’s royalties have been hit by biosimilar competition. Halozyme Therapeutics reported what it described as a “strong start to 2019” with revenues of $57 million (€51 million) – up 84% year-on-year – attributed to $30 million upfront license fee from a license agreement struck with argenx in February. That deal gives argenx access to Halozyme’s ENHANZE drug delivery technology to…