With the coronavirus pandemic disrupting nearly every part of the drugmaking industry, there's concern the ripple effect will spread to the Food and Drug Administration, too.
While the agency is focused on responding to the outbreak, it still needs to carry out the critical work of reviewing experimental medicines for other diseases.
The first quarter passed with little apparent disruption, evidenced by last week's on-time approval of Bristol Myers Squibb's multiple sclerosis drug Zeposia. But the FDA will be under immense pressure over these next few months to prove that, amid a crisis, it can maintain business as usual.
Its ability to do that will have a direct impact on all U.S. drugmakers to some extent, but especially these five, which have approval deadlines scheduled for the second quarter.
The five experimental drugs are listed in order of the currently scheduled date by which the FDA has agreed to make a decision on approval.
Neurocrine's opicapone for Parkinson's disease
Neurocrine Biosciences should find out by April 26 whether the FDA has approved a drug known as opicapone for the adjunctive treatment of certain patients with Parkinson's disease.
In Europe, opicapone has been approved for this use since mid-2016, and is sold by BIAL Pharmaceuticals under the brand name Ongentys. About eight months after the approval, BIAL gave Neurocrine rights to develop and commercialize the drug in the U.S. and Canada in exchange for $30 million upfront.
Though analysts expect an approval in the U.S., they don't see opicapone having a huge impact on Neurocrine's bottom line — at least in the near-term.
Still, diversification could help Neurocrine.
The San Diego-based biotech gets almost all its revenue from Ingrezza, a drug that treats the involuntary movements that can arise after years of taking psychiatric medications. Ingrezza sales have leaped each year since its 2017 approval, and, according to RBC Capital Markets, the consensus among analysts is that the drug will hit $1 billion in sales this year.
Yet, as with many other drugs, the novel coronavirus could have an effect on Ingrezza's growth.
In a March 26 note to clients, RBC analysts wrote how Neurocrine is "likely to be more negatively impacted" by the virus than other biotechs, partly because of Ingrezza. RBC explained that the drug relies heavily on in-person visits between sales staff and doctors to drive prescriptions for new patients. That's a challenge, given Neurocrine recently transitioned its field sales force to remote work.
Bristol Myers Squibb's Opdivo plus Yervoy for lung cancer
For a majority of patients with advanced non-small cell lung cancer, combining immunotherapy with chemotherapy has become a go-to option for initial treatment.
Thus far, it's been Merck & Co. and Roche that hold approvals to use their immunotherapies as part of first-line combination drug regimens. Bristol Myers Squibb, which was once ahead of rival lung cancer drugmakers, fell behind due to a series of setbacks and clinical trial failures.
A key part of Bristol Myers' bounce-back strategy has been an in-house combination of the immunotherapies Opdivo and Yervoy. The combination is now approved for certain patients with colorectal, skin, kidney, or liver cancers, but the pharma has had a difficult time proving the pairing in lung cancer.
Aiming to find a niche, Bristol Myers focused on people whose tumors have unusually high levels of genetic mutations, a measure known as tumor mutational burden, or TMB. But that marker is relatively new and not as established with regulators as another immunotherapy biomarker, called PD-L1. Bristol Myers has had to pull approval applications in both Europe and the U.S. over the past two years to gather more data to support its work with TMB.
Though Bristol Myers has since given up on approval in Europe, it could finally have its day in the U.S. on May 15, when the FDA is supposed to make a decision on the use of Opdivo plus Yervoy in lung cancer. Whether it could compete is another question.
Roche's risdiplam for spinal muscular atrophy
Treatment for spinal muscular atrophy, a rare, potentially deadly genetic disease often referred to as SMA, has significantly changed over the past few years. Doctors, for years lacking an approved medicine, can now prescribe two therapies that, for many patients, appear to slow the progressive weakness which characterizes their condition.
This quarter could bring a third option in risdiplam, an oral medicine from Roche and PTC Therapeutics that, like Biogen's Spinraza and Novartis' Zolgensma, boosts the production of a protein that SMA patients don't have. The FDA should decide by May 24 whether to approve Roche's drug.
The key potential advantage for risdiplam over the other approved medicines is convenience, as patients could receive risdiplam without having to go into a doctor's office like they do with Spinraza and Zolgensma, which are both infused. This was already a boost for Roche in a pre-coronavirus world, and may be more so now that traveling for treatment poses additional risk.
Risdiplam could also look like a more favorable option for patients who have trouble tolerating the spinal taps that accompany Spinraza, or who might not be eligible for that drug or Zolgensma.
Spinraza and Zolgensma each came to market with extremely high price tags, and have since become sales drivers for their respective developers. Investment banks have similarly high revenue estimates for risdiplam.
But the drug's commercial fate will depend, in part, on whether the FDA grants it a broad label, as the agency did for Spinraza in 2016. Roche has been testing the drug on newborns to 60 year olds with multiple forms of the disease.
Novartis' ofatumumab for multiple sclerosis
Ofatumumab has been on the U.S. market for over a decade, sold as Arzerra for the treatment of a specific type of leukemia. The drug was developed by GlaxoSmithKline and Denmark-based Genmab, though Novartis acquired GSK's rights several years ago.
Now, Novartis is aiming to expand the drug's label with an approval for relapsing multiple sclerosis. Supporting that mission are data from two Phase 3 studies which showed patients given ofatumumab experienced significant reductions in their number of relapses compared to those given Aubagio, Sanofi's pill for multiple sclerosis.
While companies often caution against comparing results from different trials, Novartis' data appeared to show ofatumumab having a similar level of benefit as Roche's Ocrevus, a drug that quickly shot to the forefront of multiple sclerosis treatment after its 2017 approval.
Ocrevus is just one of the roughly 20 disease-modifying drugs that ofatumumab would go up against should it gain approval, scheduled to be decided by June. Other competitors include Biogen's Tecfidera, Sanofi's Lemtrada and Bristol Myers' Zeposia, which was approved this month. There's also Gilenya, Novartis' own blockbuster pill, as well as a relatively cheap option in the generic version of Teva's Copaxone.
The market is likely to grow more crowded too, as Johnson & Johnson just filed for approval of a drug called ponesimod.
Intercept's obeticholic acid for NASH
Investment banks see much at stake in a fatty liver disease called nonalcoholic steatohepatitis, or NASH for short.
At earlier stages, NASH can be effectively treated with diet and exercise. When it progresses too far, however, it can lead to liver cirrhosis and the need for a transplant. Data on the size and severity of the NASH population is fragmented, but estimates hold that millions of people have NASH in the U.S. alone.
Though NASH is a focus for many drugmakers, none have been able to usher a medicine to market. Intercept Pharmaceuticals, though, could change that with a drug called obeticholic acid, which scored an industry first by succeeding in a late-stage study of patients with moderate to severe NASH.
That milestone aside, the results were far from perfect. They'll be front and center at a June 9 meeting where Intercept and FDA staff debate whether the drug is worth approving. The agency is set to make an approval decision by June 26.
An approval in NASH could be a boon to Intercept's sales, yet it would also put the company in the tricky position of being the first to navigate pricing, reimbursement and commercialization. CEO Mark Pruzanski believes his company can do just that, saying in January he felt "confident that we will be ready and we will be able to drive the successful launch of this drug."