[Updated 12/22/17, 2:37 p.m. See below.] This year is wrapping up as one of the strongest on record for FDA drug approvals. As of this morning, the regulator has approved 46 novel drugs in 2017—a total that beats the mark set two years ago. With one week left in the year, the FDA could add even more drugs to the list.
The totals reflect “new molecular entities,” compounds that have not been approved by the FDA before, either alone or in combination with other products. Regulatory Focus notes that the tally of 46 approvals this year does not include the CAR-T cancer treatments from Novartis and Kite Pharma, which are made by engineering a patient’s immune cells. Nor do they include gene therapy, such as the Spark Therapeutics’ treatment for a rare form of blindness, which the FDA approved this week. The drug approval that put the FDA over the top, La Jolla Pharmaceutical’s (NASDAQ: LJPC) Giapreza, is made from a synthetic form of the hormone angiotensin II. The flurry of approvals suggests that commissioner Scott Gottlieb is trying to make good on his pledge to bring new drugs to the market as quickly and efficiently as possible. [Post updated to reflect FDA’s latest drug approval totals.]
In other news this week, Congress passed a tax overhaul bill, a life sciences venture capital firm brought in its biggest ever cash haul, and in a move that probably would make the late comedian George Carlin smile, the Centers for Disease Control and Prevention reportedly revealed a list of seven banned terms. Let’s get to these stories and more in this week’s roundup.
—The Republican tax rewrite is a done deal, with groups and individuals across the country scrambling to assess its impact. Like all other industries, the life sciences will get a corporate tax cut. But we still don’t have a federal budget. Late Thursday, Congress passed a measure that punts the deadline for agreeing on a budget to Jan. 19.
—It’s unclear what future budgets will hold to combat the opioid crisis. The Centers for Disease Control and Prevention released 2016 data that showed the rise in opioid overdose deaths—more than 42,000 last year—continues to lower U.S. life expectancy. President Trump declared the crisis a health emergency in October but no new funding has been allocated for the fight.
—Late last Friday the Washington Post reported that CDC officials told scientists to leave out seven words, including “transgender,” “science-based,” and “fetus,” from budget proposals. The CDC pushed back, saying it wasn’t a ban, just suggestions for discussions about “budget formulations”—that is, the need not to offend conservative lawmakers who hold the agency’s purse strings.
—Pfizer (NYSE: PFE) unveiled plans for the boatloads of extra overseas profits it can now repatriate to the U.S., thanks to the tax bill. The drug giant announced a $10 billion stock buy-back and raised its quarterly dividend.
—The federal Government Accountability Office released a report on pharmaceutical profits. From 2006 to 2015, industry revenues rose about 50 percent. Two-thirds of companies increased their profit margin, with the top 25 companies averaging between 15 and 20 percent. (The world’s largest non-drug companies averaged between 4 and 9 percent.) Pharma’s gains were not matched by R&D spending, however. From 2008 to 2014, it rose slightly, from $82 billion to $89 billion.
APPROVALS & ASSESSMENTS
—Spark Therapeutics (NASDAQ: ONCE) won FDA approval for its gene therapy to treat a rare, inherited form of blindness, but the company is waiting until January to reveal how much it will charge for the drug.
—Roche’s Genentech division notched FDA approval for pertuzumab (Perjeta) combined with trastuzumab (Herceptin) and chemotherapy to treat HER2-positive breast cancer after surgery, despite only a modest benefit over trastuzumab and chemo alone in keeping patients cancer-free. FDA also gave full approval to the pertuzumab combination in patients before surgery.
—Having completed an FDA application for what could be the first RNA interference medicine on the market, Alnylam Pharmaceuticals (NASDAQ: ALNY) and partner Sanofi (NYSE: SNY) have now filed for approval of the drug patisiran in Europe as well.
—The FDA approved netarsudil (Rhopressa), a glaucoma drug from Aerie Pharmaceuticals (NASDAQ: AERI). But the drug’s commercial prospects are unclear due to questions about the drug’s tolerability and effectiveness relative to other therapies.
—Loxo Oncology (NASDAQ: LOXO) also began submitting paperwork to the FDA for larotrectinib, a drug being developed for tumors that have a genetic alteration known as a TRK—regardless of what tissue they originated in.
—An Alzheimer’s disease drug that Biogen (NASDAQ: BIIB) and Eisai are testing fell short of its goal, according to interim results from a mid-stage study. But the partners plan to complete the trial, which they hope will show better results.
DOLLARS & DEALS
—Cambridge, MA, biotech Aura Biosciences raised a $30 million Series C to continue developing a light-activated nanoparticle therapy for the rare eye cancer, ocular melanoma.
—London biotech Orchard Therapeutics, which is developing gene therapies for a variety of rare diseases, raised a $110 million Series B round.
—Ultragenyx Pharmaceutical (NASDAQ: RARE) sold a priority review voucher to Novartis (NYSE: NVS) for $130 million, cash that the Novato, CA-based company plans to apply to its rare disease drug pipeline.
—Biogen and Ionis Pharmaceuticals (NASDAQ: IONS), which teamed together to develop the first approved therapy for spinal muscular atrophy, nusinersen (Spinraza), joined up in a new alliance to develop additional drugs for SMA.
—Cambridge, MA-based Flagship Pioneering hauled in $618 million, its biggest capital raise ever, to back a new crop of biotech startups.
MOVERS & SHAKERS
—This week we recapped our latest life sciences event, “New York Biotech: The Future is Now.” Check out this piece for some of the photos and main topics of conversation. And read this Q&A with Vicki Sato to hear her perspective on how to grow New York’s life sciences community.
Alex Lash and Ben Fidler contributed to this report.