FDA, kickin’ butt and taking orphan names

A recent blog by FDA commissioner Scott Gottlieb outlines the progress FDA has made eliminating the backlog of ~200 orphan drug designation requests. Recall, an orphan or rare disease is one that “generally affects fewer than 200,000 people in the United States.” But these orphans aren’t cute. They are big business. According to EvaluatePharma “worldwide orphan drug sales are forecasted to total $209bn in 2022.” According to Gottlieb, the number of orphan designation requests more than doubled between 2012 and 2016, rising to 568 new requests. FDA plans to “close a loophole that allows sponsors to avoid an obligation to study drugs in pediatric indications.” The Pediatric Research Equity Act (PREA) was enacted to promote more pediatric studies. Hasn’t happened but Dr. G. plans to change that. Bravo.

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It’s aliiive! And it’s approved!

Last week, the FDA approved Novartis’ Kymriah—a “living drug” that works by making immune cells realize they should get rid of those pesky leukemia cells making a mess of things. We’ve been following this story since June, because it’s really cool for a couple reasons. First, it marks the first time a gene therapy has been approved for use in the US, although more CAR-T treatments are in the pipeline. Second, the treatment is designated for the most prevalent form of childhood cancer in the US—acute lymphoblastic leukemia (ALL). There is a bit of worry that the treatment is prohibitively expensive, which is what sunk the first gene therapy approved in the EU, but maybe competition will help drop prices.

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Regulators gonna regulate

This writer is confused. Is the government in the business of deregulating drug use (i.e., marijuana, i.e., the sticky icky) or regulating drug use? The FDA has announced its desire to lower the amount of nicotine in cigarettes in an effort to decrease addiction, reduce smoking, and improve health. At the same time, The Washington Post reports that the FDA is delaying its regulation of trendy smoking alternatives such as e-cigarettes, hookah, and vape thingies that all the cool kids are using. The hope is that keeping these smoking alternatives easily available and less regulated will give smokers ready access to “safer” alternatives to taking in their nicotine. Here is a list of the 20 most destructive drugs, as reported in The Economist. Yikes.

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Patient safety? Nah I’m good

When a medical device malfunctions, it is up to the doctor to alert the hospital, the hospital to alert the device maker, and the device maker to alert the FDA 30 days later. Not exactly a fast process. Well the US House of Representatives just approved a renegotiated agreement between the FDA and device industry that would push this 30 days back to three months. While device makers are happy because this agreement also includes lowering the proposed annual user fees, it has some watchdog groups and critics up in arms and bringing up all the bad memories the device industry wishes they could forget. Who knows, maybe malfunctioning trends will become more apparent with this extension, but it certainly doesn’t scream “patient safety.”

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It’s not you, it’s me…

…are the words you really never want to hear, unless you are Amicus Therapeutics. In a rare reversal, the FDA, under the leadership of Scott Gottlieb, told Amicus Therapeutics they didn’t have to run expensive and lengthy safety studies before seeking FDA approval. Under the previous FDA leadership, Amicus was told to conduct extra safety studies, namely for GI symptoms, for their Fabry disease candidate migalastat. Many in the pharma industry are heralding this as a new era at the FDA. It’s a good time to be a biotech lately. As of press time, the iShares Nasdaq Biotechnology Index (IBB) is up 5.4% the past month, 7.1% over the past three months and 16.8% on the year. Can you hear that? That’s the sound of the VC community raising more money.

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FDA: Endo’s opioid is a no-no, has got to go

For the first time, the FDA is requesting that a drug maker remove its product from the market for public health reasons. Endo Pharmaceuticals’ Opana ER—an opioid designed to continuously manage moderate to severe pain—has already faced scrutiny for being easy to abuse via snorting. Turns out addicts aren’t too fond of the ‘extended’ aspect of the drug. To combat this, the company added a coating that made the drug harder to crush… so abusers injected it instead. Not only did this reformulation not meet the FDA’s standards of officially being abuse-deterrent, but the rise in injection abuse is also tied to an HIV/Hep C outbreak caused by needle sharing. God save us from people who mean well.

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You’re going to put that…where?

If you’ve ever dropped something down the drain, you know how hard it can be to see around the drain’s curves or retrieve an object using straight, rigid tools. Spoiler alert: You pretty much can’t. Now imagine that drain is the colon of a patient who would really prefer you didn’t use a stiff, unbending surgical instrument to prod around inside their delicate organ. Luckily for these patients, Medrobotics has received FDA approval to use its Flex Robotic System involving “steerable and shapeable robotic products” in colorectal procedures. Basically, it’s a bendy surgical robot that surgeons guide through twisty areas using a joystick. Awesome. InsightCity imagines the surgeons will have fun reliving Nintendo’s glory days.

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