The advisory committee route for approving a first biosimilar to a reference product is no longer necessarily the norm, going by FDA's recent approval of Mylan/Biocon's pegfilgrastim-jmdb, which references Amgen’s Neulasta; agency’s view that a meeting was not needed should encourage other biosimilar aspirants.

Medicines Development for Global Health is the first nonprofit to earn a priority review voucher for bringing a neglected tropical disease therapy to US approval. Project carried on research from the Wyeth era.

A new service provided by UK HTA body NICE for evaluating economic models that will be reviewed by health technology appraisal bodies has been a success, according to Takeda.

Internal group headed by Principal Deputy Commissioner Rachel Sherman will develop implementation recommendations, which could include new guidance, regulations, a Q&A document or ‘nothing at all,’ Commissioner Gottlieb tells BIO annual meeting; he promises consultation with the law’s supporters and critics on Capitol Hill ‘to fully understand what the intent was with different provisions.’

The four latest products to be accepted onto the European Medicines Agency’s popular priority medicines scheme target mantle cell lymphoma, chikungunya, hematopoietic stem cell transplant patients, and the rare monogenic disease, X-linked myotubular myopathy.

Predictive Toxicology Roadmap will help make drug development more efficient by promoting new technologies for safety testing, Commissioner Gottlieb says.

FDA alerted Mylan to expect a complete response letter related to its ANDA for generic Advair. The company said it still could receive an approval prior to the standard 90-day time period after responding to the letter, because the application has a priority designation.

Two CEOs explain why their rare disease companies will not grant preapproval access to investigational drugs under new federal law; Alnylam sees no need for framework beyond FDA’s expanded access pathway, while Sarepta is focused on the ‘grand goal’ of getting drugs studied and approved for the patient community.

US FDA to head off cell/gene therapy manufacturing issues with pre-IND meetings; Republic of Ireland will “chase any opportunities” for business partners stemming from Brexit, even while hoping UK reconsiders EU departure; AMAG won’t follow the flibanserin PR approach with newly submitted female sexual dysfunction drug bremelanotide; United Neuroscience CMO Ajay Verma says Alzheimer’s trial setbacks are “learnings,” with progress toward novel therapies moving faster than it has for cancer and HIV.

US FDA touts new evidence that high performance of pharmaceutical manufacturing sites on 10 quality maturity metrics correlates with robust pharmaceutical quality systems, operational excellence and good business. An FDA-funded study by St. Gallen also finds that low performance, on the other hand, correlates with manufacturing problems.

Generic industry officials ask that information requests and discipline review letters be issued during ANDA amendment assessments, but the US agency says they are not allowed in GDUFA II.

The potential of an impending “apocalypse” of antibiotic drug shortages means that the pharmaceutical industry should pay careful attention to managing fragile supply chains for these drugs. A report details how manufacturers can manage these supply chains to avert potential shortages.

Energy & Commerce's Republican and Democrat leadership ask FDA Commissioner Scott Gottlieb about work by the agency's Office of Criminal Investigations focused on opioid drugs, the office's staffing and its priorities. They note FDA's FY2018 appropriation included $94m for its work in international mail facilities.

Target date moved from August 2018 to February 2019 for publishing an NPRM for allowing currently Rx drug ingredients to be available nonprescription under conditions of safe use. US HHS annual spring update on regulatory priorities for FDA and its other agencies also pushes back target dates for other OTC drug NPRMs, including FDA's long-delayed finalization of several monographs including sunscreen ingredients.

FDA issued 39 warning letters to OTC drug facilities, most of them outside the US, in 2017, a number significantly boosted by an initiative to inspect a total of 1,000 foreign drug manufacturers from January 2017 through September 2019 , says CDER compliance director Donald Ashley. Many GMP violations behind FDA's warning letters concerned “rudimentary" requirements.