Global Markets

America has 20 approved biosimilars but you’ll never believe how many have launched!

With the FDA approving Amgen And Allergan’s version of Roche’s Herceptin (trastuzumab) America’s biosimilar tally has reached 20… But only seven have reached the market. Clickbait headline aside, this week marks another major milestone in the US biosimilar market: the approval of its twentieth biosimilar Kanjinti (trastuzumab-anns), a version of Roche’s cancer monoclonal antibody Herceptin developed by Amgen and Allergan. The approval is the third in Amgen’s portfolio and the second of four biosimilars from an Amgen and Allergan collaboration…

Hitachi supplying gene therapy Zynteglo for bluebird in EU

bluebird bio will use Hitachi Chemical subsidiary apceth Biopharma to commercially manufacture its recently EU approved gene therapy Zynteglo. Earlier this month, the European Medicines Agency (EMA) approved orphan drug Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) – previously known as LentiGlobin – as a one-off treatment for beta-thalassaemia. Zynteglo uses a lentiviral viral vector to add functional copies of a modified β-globin gene into a patient’s own stem cells, addressing the underlying genetic cause of patients for whom a…

Ionis pledges to up its RNA game in SMA and elsewhere

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…

Vaccines the ‘backwaters of the pharma industry’ due to low price expectation

Historic low prices and the fact they are administered to healthy people means vaccine development plays second fiddle to biologics, according to a panel of experts. At the BPI West event in Santa Clara, California earlier this year, Rahul Singhyi, COO of Takeda vaccines, told delegates that vaccines used to be viewed as “the backwaters of the pharma industry.” Fast forward several months to the BPI Europe event in Vienna, Austria, and Bioprocess Insider asked four more experts within the…

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…