Industry news

Drug pricing system broken: Where do we go from here?

Mari Serebrov / BioWorld

When it comes to how drugs are priced in the U.S., everyone seems to agree on one thing: The system is broken. But before they can fix it, Congress and policymakers need to first understand how it is structured now and where the problems are. The trouble is, they don't know that they don't get it. That much was clear in a House oversight hearing Wednesday on the pricing of Mylan NV's Epipen (epinephrine), which was a missed opportunity to shed some light on a very murky subject.

29 September 2016

Ganaxolone Reduces Seizures in Females with PCDH19 Pediatric Epilepsy

Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced top-line results from a Phase 2 exploratory open-label clinical trial evaluating ganaxolone, its CNS-selective GABAA modulator, in females with PCDH19 pediatric epilepsy. In the trial, ganaxolone reduced seizure frequency from baseline in the majority of patients enrolled in the study and was generally safe and well tolerated. PCDH19 pediatric epilepsy is a rare, serious epilepsy characterized by early-onset cluster seizures, cognitive and sensory impairment, and behavioral disturbances, with no approved treatments. In 2015, the U.S. Food and Drug Administration granted Orphan Drug Designation to ganaxolone for the treatment of PCDH19.

US and Europe Collaborate on Treatment of Rare Diseases

BioPharm International

The European Medicines Agency (EMA) announced on Sept. 26, 2016 that the EMA/FDA cluster, or working group, on the treatment of rare diseases had its first meeting on Sept. 23, 2016. The cluster was created to share best practices between the US and EU agencies and “provide a forum for confidential exchange of draft documents, policies under development, and more detailed information supporting the scientific basis for decision making on medicine development,” according to an EMA press release.

Pfizer Decides to Remain One Company

BioPharm International

On Monday Sept. 26, 2016, Pfizer announced its decision to remain as one company. The company had been debating whether or not to divide Pfizer Innovative Health and Pfizer Essential Health into two, separate publicly traded companies. On Monday, leadership decided against this strategy noting Pfizer is best positioned to maximize shareholder value in its current state.

Separating hope from hype in the latest cancer treatment ‘revolution’

Damian Garde / STAT

There’s new hope that a much-hyped class of immunotherapies could revolutionize cancer treatment: Data released this week from an early test on patients stirred hope that the first such therapy could hit the market soon. But experts remain cautious about safety and efficacy — and warn that big questions remain.

For Pfizer, Bigger as One May Be Better Than Smaller With Two

Jared S Hopkins, Cynthia Koons / Bloomberg

By deciding not to split in two after a lengthy evaluation, the New York-based drugmaker is keeping greater leverage with the health insurers and pharmacy benefit managers that have gained increasing power since it first considered a breakup years ago.

Ophthalmology’s diverse pipeline could rejuvenate previously stagnant therapy area, says GBI Research

Global Business Intelligence Research

The company’s latest report states that scientific advancements in this therapy area – which includes glaucoma, age-related macular degeneration (AMD), diabetic macular edema, diabetic retinopathy and dry eye syndrome – have revealed a broad range of novel potential molecular targets in recent decades, enabling progress in a previously stagnant field. This is reflected by the high number of products in development, with 782 products currently in the ophthalmology therapy pipeline.

Asia-Pacific gastric cancer market set to experience strong growth to $2.7 billion by 2022, says GBI Research

Global Business Intelligence Research

The Asia-Pacific (APAC) gastric cancer market is set to rise from $1.3 billion in 2015 to $2.7 billion by 2022, at a strong compound annual growth rate of 11.4%, according to business intelligence provider GBI Research.

Drugmakers agree to clean up plants, take other steps to fight antibiotic resistance

Eric Palmer / Fierce Pharma

More than a dozen pharmaceutical companies from Big Pharma players to CMOs have agreed to take steps to fight antibiotic resistance, including reviewing their manufacturing and supply chains and working on standards for cleaning up antibiotic waste discharge.

Australia gears up to reform drug, device regulations, speed access to therapies

Tamra Sami / BioWorld

The pharma and medical device industry praised the Australian government on new reforms announced late last week that increase the pathways for marketing approval for drugs and devices and reduce regulatory red tape and redundancies.