In this week’s news, US FDA issues guidelines for ultra-rare disease therapies, Ireland rejigs parallel import rules and more.

US issues new production guidelines for gene therapies for ultra-rare diseases

The US FDA has published more guidelines for so-called ‘N of 1’ gene therapies for ultra-rare diseases, including information for manufacturers.

The US regulator issued the CMC guideline this week, setting out how companies making antisense oligonucleotide drugs should go about doing so.

Specifically, the agency expects developers to provide enough CMC information “to ensure the proper identification, quality, purity, and strength of the investigational drug product.” 

HPRA has imposed new rules for parallel imports

The Health Products Regulatory Authority (HPRA) will only grant a parallel product authorization (PPA) if the imported product in question is authorised in Ireland under a revision introduced this week.

The rule change – which also stipulated that the imported product has the same API in the same form as the approved product – is the first modification of the PPA legislation since it was introduced in 2017.

Industry wants more info on US FDA safety reporting draft guidance

Industry group PhRMA and AdvaMed want the US FDA to clarify its draft guidance on safety reporting for investigational drugs and devices.

The US regulatory published the comments this week.

Also in the news

Endpoints visited AstraZeneca’s newly operation R&D facility in Cambridge, Massachusetts.

BeiGene has purchased a 42-acre site at the Princeton West Innovation Campus in Hopewell, N.J. to house a new manufacturing campus and clinical R&D centre.

Scrip covered rumours surrounding the VMIC facility. The tax payer-funded plant was intended to make the UK self-sufficient for vaccines, however, there is speculation it will be sold off to a private buyer.

DCAT reported that the US FDA has updated its guidelines for nitrosamine, the API linked to several incidences of cancer in recent years.

Merck, which is known as MSD outside the US, has acquired Acceleron Pharma, the developer of a drug called sotatercept that could become the first disease-modifying treatment for pulmonary hypertension.

CDMO trends

Some developments of note in the contracting sector:

IPS-Integrated Project Services will design a new high speed filling line being installed at the plant Jubilant HollisterStier is building in Spokane, Washington.

Lonza has invested in additional microbial development capabilities in Switzerland to support demand for microbial-derived proteins.

GreenLight Biosciences has hired Samsung Biologics to manufacture its messenger RNA COVID-19 vaccine candidate at commercial scale.

National Resilience has bought SwiftScale Biologics, a San Leandro, California-based company developing cell-free protein synthesis (CFPS) technology.

WuXi Biologics says it has completed the first GMP production run using its new 24,000L line at its drug substance facility, called MFG5.

Sartorius is investing around 270 million euros to expand its activities in Songdo in Incheon, South Korea. The company plans to launch cell culture media production and to assemble sterile systems at the site.