Regulations

bluebird opens gene therapy plant as EMA backs gene therapy

The EMA has recommended approving Zynteglo, bluebird bio’s one-off treatment for beta-thalassaemia. Meanwhile the cell and gene therapy firm has cut the ribbon on a 125,000-square-foot facility in Durham, North Carolina. The EMA’s Committee for Advanced Therapies (CAT) recommended Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) – also know as LentiGlobin – today, paving the way for bluebird bio to bring its gene therapy product to market. The product uses a lentiviral viral vector to add functional copies of a…

Biogen stock plunges $18bn on Alzheimer’s MAb failure

Biogen lost around 30% of its value after discontinuing a Phase III trial of Alzheimer’s disease candidate aducanumab. The CRO involved will also take a hit in revenues. Codeveloped with Eisai, aducanumab had been undergoing Phase III clinical trials in patients with mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease dementia. But the firms took the decision to halt the study this week – along with the EVOLVE Phase 2 safety study and the long-term extension of…

Two Form 483s not indicative of systemic manufacturing issues, Biocon

Biocon has received two US FDA 483s with two and six observations at a drug product plant and an insulin API plant, respectively. The first 483 came following a pre-approval inspection of a new injectable manufacturing line for a biologic drug product at the firm’s site in Bangalore, India conducted between February 7 and 15. The Form has since been published by the US Food and Drug Administration (FDA) and the two observations relate to issues with microbial prevention procedures…

Biosimilar… but different: FDA tweaking nonproprietary name guidance

The US FDA has proposed changes to its nonproprietary naming guidance to remove the proposed controversial four-letter suffix from older biologics. In 2017, the US Food and Drug Administration (FDA) published guidance to include a random four-letter suffix to the international nonproprietary names (INN) of all biological products. The ruling essentially distinguished a biosimilar from its reference product, so Amgen’s reference product Neupogen, for example, is known as ‘filgrastim-jcwp,’ while Sandoz’s biosimilar version Zarxio – the first biosimilar approved and…

Precision Bio $100m IPO to support allo CAR-T build-out

Precision Bio hopes the initial public offering (IPO) will advance its off-the-shelf CAR-T candidates, based on its ARCUS genome editing platform. North Carolina-based Precision Bio has applied for a listing on the Nasdaq exchange. According to its Form S-1 filed with the SEC, the firm will use the anticipated $100 million (€88 million) raised in the IPO to advance and expand its clinical and preclinical development programs. This includes plans to complete a Phase I/IIa clinical trial for its CD19…

FDA commissioner resignation ‘a great day for rogue stem cell clinics’

Scott Gottlieb will step down from his position as commissioner of the US FDA next month. He issued more cell and gene therapy guidance in his two-year tenure than had appeared in the previous ten. In May 2017, the US Senate confirmed Dr Scott Gottlieb as commissioner of the US Food and Drug Administration (FDA). This week, nearly two years on, and Gottlieb announced he is to step down from the role in the next month. Among Gottlieb’s achievements are…

Under fire, BMS insists $74bn Celgene deal ‘next step in proven strategy’

Bristol-Myers Squibb has dismissed claims by investor groups that its proposed acquisition of Celgene “is poorly conceived and ill-advised.” In January, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to buy Celgene for $74 billion (€65 billion). But this week Starboard Value LP, which holds a stake of 1.63 million shares in BMS, criticized the deal in a letter to fellow shareholders. “We believe that Bristol-Myers is deeply undervalued and the recent announcement of the Company’s proposed acquisition…

Immunomedics: Data integrity and quality issues cited in FDA 483

The US FDA found antibody-drug conjugate developer Immunomedics to have manipulated data during an inspection last year. In January 2019, the US Food and Drug Administration (FDA) issued a complete response letter (CRL) to Immunomedics for its antibody-drug conjugate (ADC) candidate sacituzumab govitecan, being developed to treat breast cancer. The firm said at the time: “The issues related to approvability in the CRL were exclusively focused on Chemistry, Manufacturing and Control matters and no new clinical or preclinical data need…

FDA guidelines aim to improve quality and lower costs

The US FDA has issued draft guidance intended to promote innovation in pharmaceutical development and manufacturing. Entitled ‘Program for the Recognition of Voluntary Consensus Standards Related to Pharmaceutical Quality Guidance for Industry,’ the draft guidance has been produced by the US Food and Drug Administration (FDA) to informally recognize voluntary consensus standards relating to pharmaceutical quality for products under the jurisdiction of the Center for Drug Evaluation and Research (CDER). “The overall goal of this program is to promote development…

Phacilitate: Cell & gene industry flocks to Miami

The cell and gene therapy industry congregated in Miami to discuss supply chain strategies, regulatory rationale and the high manufacturing costs of advanced therapies. BioProcess Insider attended the Phacilitate Leaders World Summit, co-located with the World Stem Cell Summit, in Miami, Florida last week. The conference reflected on the first full year where three advanced therapies – Kymriah, Yescarta, and Luxturna – proved themselves on the commercial stage, heralding in a new frontier for regenerative medicines. But industry quickly rose above…