The Readout Damian Garde & Meghana Keshavan

The FDA is trying to make gene therapy companies compete

The rise of gene therapy promises one-time treatments for devastating diseases. It also threatens payer budgets with seven-figure price tags. Now the FDA, powerless to actually regulate drug prices, is trying to clear the way for competition that might make gene therapy more affordable.

As STAT’s Nicholas Florko reports, the agency finalized a slew of new policies yesterday with the goal of streamlining the process of getting a gene therapy approved. That means standardizing the rules on manufacturing and, perhaps most important, determining whether competing gene therapies qualify for FDA special treatment.

“We’re very acutely aware that if we want to see [gene therapies] come to benefit more patients were going to have to see the price come down,” Peter Marks, the director of FDA’s biologics center, told STAT. “And towards that end, that’s why we want to do we can to float all boats.”

Read more.

Speaking of gene therapy, is it a good business?

While the public discussion of gene therapy tends to focus on how much they cost, the anxiety in biotech circles is a bit different. The products are expensive to make, complicated to administer, and usually treat tiny patient populations. Is anyone going to make money on these things?

We’ll get a useful hint this morning from Novartis, which is slated to report its earnings and by extension the sales for Zolgensma, a gene therapy for spinal muscular atrophy. Novartis’s product quite famously costs $2.1 million for a one-time dose, and it has also shown an unprecedented benefit for children with the most severe form of SMA.

On paper, Zolgensma’s combination of efficacy and convenience would make it the treatment of choice for some SMA patients, preferable to Biogen’s Spinraza, which is taken every few months for life and costs about $375,000 a year. But in practice, getting the health care system on board with a large one-time cost might prove difficult. 

And thus the commercial fate of Zolgensma will be keenly watched by the likes of Bluebird Bio, BioMarin, Audentes and anyone else hoping to make a profit on one-time treatments.

Where are all the woman-founded biotech companies?

If the women of MIT’s science and engineering departments founded startups at the same rate as their male peers, the past decade would have produced about 40 more biotech companies than it actually did. In an effort to figure out what’s behind that gender disparity, three prominent MIT scientists spent a year collecting data and grilling stakeholders, and they’ve come up with a first step toward making things more equitable.

As STAT’s Sharon Begley reports, that means starting at the board level. The trio has convinced five major venture firms “to do all in [their] power” to make sure the boards of their portfolio companies are 25% female by the end of 2022. The working theory is this: Women can’t start companies if they can’t get funding, and VC tends to be a who-you-know business, so putting more women on boards should help create a virtuous cycle of entrepreneurship.

“Service on a board creates access to a network of investors, leading academic scientists, and other key leaders,” said Amy Schulman, a partner at Polaris Ventures who serves on several biotech boards. “It’s a door opener and a credentialer.”

Read more.

Biogen’s big unanswered question

The world has had plenty of time to chew over Biogen’s promise to submit  a once-waylaid Alzheimer’s disease treatment for FDA approval. The question now is simply when.

And we might get an answer tomorrow, when Biogen reports its quarterly earnings. To date, executives have promised only that they’d submit the drug, aducanumab, as soon as possible, which analysts have taken to mean the first quarter. Any detail Biogen provides will be key to setting expectations for what could be a superlatively lucrative product.

As Baird analyst Brian Skorney pointed out this week, timing could be key. Biogen would have to submit aducanumab no later than May to ensure that the FDA’s final decision would come ahead of the 2020 presidential election, according to Skorney. The company hasn’t been a campaign topic thus far, but there’s a mounting concern among investors that aducanumab’s debatable efficacy — and undoubtedly controversial price — could become a political issue that sways the FDA.

Speaking of earnings, STAT’s Adam Feuerstein rounded up the major quarterly themes for companies including Alexion Pharmaceuticals, Amgen, and Vertex Pharmaceuticals. Read more.

More reads

  • Prosecutors identified ‘Pharma Co. X’ as central to alleged opioid kickback scheme. Was it Purdue? (STAT)
  • Pfizer Drops by Most Since July as Sales of Key Drugs Disappoint. (Bloomberg)
  • ‘If you want to develop a new medicine, Boston is the place.’ (Boston Globe)
  • Biotech can — and must — reshape the pricing debate and protect future innovation. (STAT)

Thanks for reading! Until tomorrow,

Wednesday, January 29, 2020


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