PTC Therapeutics is one of many companies in the life sciences sector whose regulatory targets have been adversely affected by the coronavirus pandemic.

Amid the growing threat posed by variants of the coronavirus, the European Commission has published new rules allowing modified vaccines to be approved more quickly. The UK has identified one version of the B.1.167 virus variant as the most transmissible, although the jury is still out on its ability to evade vaccine protection.

 Advicenne withdrew its orphan drug designation application for Sibnayal over concerns that a key study was not designed to compare the product to standard of care.

Following a memorable cancer drug advisory panel meeting where FDA explained how accelerated approvals may be more at risk due to changing standard of care, ChemoCentryx’s ANCA-vasculitis treatment runs into trouble because of another drug’s changing indication during their Phase III study.

AstraZeneca and Novavax have not filed EUAs for their vaccines, and they never might. The pathway remains viable in the short-term, but as BLAs are approved and supplies are no longer short, the window may close.

Emergency use authorization in 12-15 year-olds follows announcement of a June advisory committee meeting on pediatric data requirements, including the FDA’s criteria for younger children. Authorization for US adolescents has raised ethical questions about prioritizing this lower-risk group over hard-hit populations overseas.

 Advicenne withdrew its orphan drug designation application for Sibnayal over concerns that a key study was not designed to compare the product to standard of care.

Anna Kaltenboeck joins the committee from Memorial Sloan Kettering’s health policy group, which has been an active participant in US drug pricing reform policy development and a critic of the biopharma industry, often through public commentary by its director, Peter Bach.

Horning describes challenges of moving from tenured professor to industry executive in accepting the award from the Healthcare Businesswomen’s Association. She is recognized for her ‘epic tenacity’ in advancing medicines and commitment to patients. Pfizer’s Rod MacKenzie receives honorable mentor award.

 Advicenne withdrew its orphan drug designation application for Sibnayal over concerns that a key study was not designed to compare the product to standard of care.

Horning describes challenges of moving from tenured professor to industry executive in accepting the award from the Healthcare Businesswomen’s Association. She is recognized for her ‘epic tenacity’ in advancing medicines and commitment to patients. Pfizer’s Rod MacKenzie receives honorable mentor award.

Authorities in China appear to be taking a tough early stance in the enforcement of a new national Biosecurity Law that took effect in mid-April, with an imported human cell shipment in Shanghai being seized. 

US FDA will ask advisory committee whether data from the trial supporting full approval of Merck’s Keytruda for second-line setting can be used to confirm clinical benefits for earlier setting of patients with locally advanced or metastatic urothelial carcinoma who are not eligible for cisplatin-containing chemotherapy following a failed confirmatory study. Genentech’s Tecentriq is also at risk of losing its accelerated approval in that space but it could buy some time as its confirmatory study is not yet complete. New competitor entrant since accelerated approvals also lessens degree of unmet need.

Similarities to AstraZeneca blood clots are clear, but more research needed before any ‘class effect’ conclusions can be drawn.

ICER President Steve Pearson expresses interest in setting drug costs for accelerated approval drugs low with price increases based on confirmatory evidence ahead of upcoming white paper from the group.

Pink Sheet reporters and editors discuss the potential for the US and other governments to waive IP protections for COVID-19 vaccines, the FDA’s inspection backlog during the pandemic, and potential reimbursement changes for CAR-T therapies.

Regulatory flexibilities such as rolling review, conditional marketing authorizations and possibly an EU emergency use authorization will play a key part in the EU’s plans to identify and approve promising new coronavirus treatments.

The pharmaceutical industry in Europe has attacked the US decision to come out in support of a relaxation of IP rights that is designed to boost global production of COVID-19 vaccines. But the move by the Biden administration could well cause other countries to rethink their opposition to the move.