The FDA is approving more drugs for rare diseases each year than ever before.

When politics, climate change or international conflicts have me down, I turn to biotech instead. There’s always a new scientific discovery, a new drug for a rare disease, a new medical device for a common ailment.

But non-industry media under-reports good news in biotech. Many biomedical stories that do go mainstream are inaccurate.

Here are ten real biomedical wins from 2018 (with a contentious #10):

  1. Alnylam* won approval for the first drug that uses RNAi, a new modality to silence mutations that cause genetic diseases like hATTR amyloidosis.
  2. Catalent won approval for a novel opioid-withdrawal treatment, a critical success near to my heart in the midst of a relentless American opioid crisis.
  3. Immuno-oncology (IO) went global. Last week, I met a teacher who’d had stage III melanoma; the 3rd patient in a Sydney trial of nivo-ipi. Melanoma killed her cousin in 2013, but she was cured after just two IO infusions.
  4. For the first time, >50% of novel drug approvals targeted rare diseases.
  5. GW Pharmaceuticals* won approval for the first marijuana-derived drug, which treats two rare, deadly forms of epileptic seizures in young children.
  6. Moderna, the top biotech unicorn (discounting Samumed), had its IPO. The Form S-1 substantively discloses Moderna’s notoriously secret work on mRNA therapeutics, a boon to other researchers in an important new field.
  7. SIGA won approval for the first drug that treats smallpox, in case the virus ever escapes its holdouts in biowarfare arsenals and BSL-4 research labs.
  8. Martin Shkreli went to prison; Elizabeth Holmes will likely follow. House-cleaning bolsters the industry’s reputation and discourages malfeasance.
  9. Amarin* scored unexpectedly positive data for a purified fish oil therapy to lower triglyceride levels, reducing often lethal cardiac events by 25%.
  10. Scientists completed the first use of CRISPR to prevent a disease in babies, a key advance despite well-justified criticism. The team lacked competency and ethics and should be sanctioned. But if the babies are healthy, with a hopefully reduced risk of HIV, the pursuit will ultimately be a net positive.

So whatever else happened in 2018, the biomedical endeavor continues its steady progress, to the benefit of us all.


*Disclosure: I’ve owned shares in starred companies within the past 12 months.

Principal at RA Capital, on the boards of 7 biotech companies. Founder/CEO of Nivien Therapeutics. Australian-American. 50 countries and counting.

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