What crisis? Zafgen fiddled while its stock burned
October 14, 2015 | FDA, Litigation, Patient Safety, Research & Development
By Ed Silverman

File this under ‘What were they thinking?

Two days after mounting speculation over a key clinical trial sent Zafgen stock plunging 54 percent, the little drug maker finally released a statement that relieved investors. And by the end of the day, Zafgen shares began recovering – somewhat – from their steep decline.

In its statement, the company disclosed that a patient died in its clinical study of an obesity drug in patients with a rare genetic disorder, but that the trial is continuing. To investors, this was a positive and suggested the drug did not cause the patient death. Zafgen did not confirm this, but investors were encouraged. Nonetheless, the episode is a textbook case of how not to handle the threat of bad news. Read More


A bill would allow NIH to establish a $400m private fund for rare diseases
October 14, 2015 | Drug Development, Research & Development
By Ed Silverman

In a bid to jump start drug development for rare diseases, a pair of lawmakers has introduced a bill that would allow the National Institutes of Health to establish a private investment fund. The idea is to raise $400 million from private investors and use the proceeds to develop early-stage compounds.

The NIH’s National Center for Advancing Translational Science would develop these compounds and then transfer them to the fund. In exchange, the agency would get a stake capped at 25 percent. The hope is to benefit taxpayers, patients, and investors, according to MIT professor Andrew Lo, who co-authored papers on which the legislation was based. Read More


Beyond bird poop: FDA warns drug maker over disturbing record keeping
October 14, 2015 | FDA, Patient Safety, Quality Control, Research & Development, Uncategorized
By Ed Silverman

Are the facilities at Unimark Remedies for the birds? That’s the impression one may gather after reading a warning letter the FDA issued to the company, which is based in India and supplies active pharmaceutical ingredients.

The agency noted that in March 2014 inspectors found evidence of bird poop and lizards. Still more disturbing, the FDA was much more concerned with a laundry list of problems concerning data integrity, which raised questions about the quality and reliability of the ingredients. Read More


Pharmalot.. Pharmalittle.. We’re Catching up on Clinton, Sanders and..
October 14, 2015 | Uncategorized
By Ed Silverman

Good morning, everyone, and welcome to the middle of the week. Congratulations on making it this far. If you were like us and stayed up to watch the Democratic presidential debate, you may want an extra cup of stimulation this morning. As noted below, there were no fireworks, at least when it came to the pharmaceutical industry. But as always, there is much else to track. So please feel free to dig into the tidbits we have assembled. Hope your day goes well and keep in touch...

During the Democratic presidential debate, both Hillary Clinton and Bernie Sanders listed drug makers among the enemies they've made; The National Comprehensive Cancer Network plans to provide patients with guidance about the cost of drugs used in their treatment; and Johnson & Johnson believes that tests showing the ability of Jardiance, an Eli Lilly diabetes drug, to save lives is a benefit that will likely be seen in the entire class of SGLT-2 drugs;. Read More


New York Attorney General probes Shkreli over antitrust concerns
October 13, 2015 | Antitrust, FTC, Generics
By Ed Silverman

For the second time in a week, Turing Pharmaceuticals is facing anti-trust scrutiny. The New York State Attorney General wants to know more about a controlled distribution system for its pricey Daraprim pill and the extent to which this program may thwart generic rivals. Last week, a US Senator asked the Federal Trade Commission to investigate the New York-based company over the same concern.

Daraprim is an anti-infective pill that Turing purchased this past summer for $55 million and then boosted the price from $13.50 to $750. As we noted previously, the 5,000 percent price hike triggered a new round of scrutiny of prescription drug costs. But a Turing executive acknowledged acknowledged that he would reject any orders from a generic drug maker. Read More


Most UK docs support disclosing payments from drug makers: survey
October 13, 2015 | Marketing, Research & Development, Uncategorized
By Ed Silverman

To disclose or not to disclose, that is the question. A new survey of UK health providers finds that an overwhelming majority of those queried - 87 percent - believe that payments from drug makers should be transparent. And 69 percent who currently have a relationship with at least one drug company say they have given – or are likely to give – permission to have payment information disclosed.

Nonetheless, there is still some resistance. Thirty-two percent believe it is unnecessary for drug makers to disclose payments to individual health care providers, and 26 percent feel disclosure will adversely affect medical innovation. Read More


A proposal for biosimilar reimbursement sparks outrage
October 13, 2015 | Biosimilars, Pricing & Patient Access, Research & Development, Uncategorized
By Ed Silverman

File this under ‘code red.’ A coterie of drug makers is upset with the Centers for Medicare & Medicaid Services over a proposal they fear would undermine the nascent biosimilar market in the United States. And over the past several weeks, the companies have enlisted the help of more than 70 lawmakers – Republicans and Democrats from both the House and Senate – to convince the federal agency to change its bureaucratic mind.

At issue is a code that governs how physicians would be reimbursed after they purchase and administer biosimilars. CMS wants to create just one reimbursement code for all biosimilars that a physician might prescribe, instead of the corresponding brand-name biologic. But drug makers say this will dissuade investment and make it harder to track side effects. Read More


Right to Try laws get it wrong by marginalizing FDA
October 13, 2015 | FDA, Patient Safety, Pricing & Patient Access, Research & Development
By Ed Silverman

On Sunday, California Governor Jerry Brown vetoed a bill that would have expanded access to experimental drugs — bucking a trend that is sweeping the country. These “Right to Try” laws, so far enacted by 24 states, allow people who are battling fatal illnesses to gain access to unapproved medicines. They also directly defy federal regulations.

These efforts, however, appear misguided. The laws threaten to unravel decades of patient protections, since the FDA is precluded from providing its traditional oversight. And the laws could even make it more difficult to obtain experimental drugs because of requirements not found in the existing federal program. And so far, it's not even clear if anyone has really been helped by the measures. Read More


Pharmalot… Pharmalittle… We’re catching up on drug prices, antibiotics and…
October 13, 2015 | Litigation, Mergers and Acquisitions, Pricing & Patient Access, Side Effects, Uncategorized
By Ed Silverman

Hello, everyone, and how are you today? We are just fine, thank you, as we prepare for another busy round of deadlines, interesting conversations and whatever else comes our way. This sounds familiar, yes? So to cope, we are quaffing a few cups of stimulation. Our flavor today is the appropriately seasonal Pumpkin Spice. Do join us. Meanwhile, here are some items of interest we have assembled. Have a smashing day and do stay in touch...

US prices for the world's 20 top-selling medicines are, on average, three times higher than in the UK, according to a new analysis; Nursing homes are emerging in the war on the overuse of antibiotics, which are some of the most frequently prescribed meds; and Roche will withdraw its Lariam anti-malarial drug from Ireland in July, but denied the move was linked to litigation over side effects. Read More


A Lilly cholesterol drug flops, so what are the implications? What the wags say…
October 12, 2015 | Cholesterol, Research & Development
By Ed Silverman

In a surprising move, Eli Lilly discontinued further development of a cholesterol-lowering treatment that some Wall Streeters thought would become a blockbuster medicine. A data monitoring committee determined that the drug, known as evacetrapib, simply wasn’t working and so was unlikely to meet the primary endpoint in a key late-stage clinical trial.

So what now? For Lilly, the decision places more pressure to succeed with other pipeline products, notably an Alzheimer’s drug. The news is also likely to raise doubts about a CETP drug that Merck is developing. But for other companies – notably, Amgen, Regeneron Pharmaceuticals and Sanofi – this may be a gift, since their new injectable treatments will face less competition. Read More