It’s a new year, which means another round of new drug launches is nearing the gate. This year, the ranks are chock-full of blockbuster hopefuls—from tumor-busting antibody drug conjugates to controversial homeopathics looking to change the food allergy market.
Thanks to the team at EvaluatePharma, we have assembled a list of the top 10 most-anticipated new drug launches of 2020 based on estimated global sales in 2024. And of those 10, eight could break the blockbuster barrier in four short years—putting a new batch of heavy hitters in pharma’s ranks.
First up is AstraZeneca and Daiichi Sankyo’s breast cancer conjugate Enhertu (fam-trastuzumab deruxtecan-nxki), the only entry on our list that has notched an FDA approval so far. The HER2-positive cancer therapy shrank tumors in 61% of patients in a phase 2 trial, putting its possible revenue at a chart-topping $2.4 billion in 2024, according to Evaluate—and AZ’s betting up to $7 billion on that prospect.
Rounding out the top five are drugs from Bristol-Myers Squibb, Novartis, FibroGen and Immunomedics—and the group is rife with interesting backstories. Take sacituzumab govitecan, for example: The TNBC therapy, with an estimated $1.44 billion in 2024 sales, was slapped back by the FDA in early 2019 on problems at a manufacturing plant. The fallout from that CRL included a top-level shakeup at the company—but it has since refiled and has high hopes for its FDA decision date in June.
Then there’s Novartis’ inclisiran, acquired in a much-discussed $9.7 billion pickup of The Medicines Company in December. The big question? Whether the high-priced deal can pay off, given the much-publicized woes suffered by inclisiran’s PCSK9 predecessors from Amgen, Regeneron and Sanofi.
The back of the list is populated by what are expected to be lesser performers, but they have their own interesting stories to tell. Read on to learn more about who’s on the list and why—and what might get in the way of their blockbuster hopes.
Drug: Enhertu (fam-trastuzumab deruxtecan-nxki)
Companies: AstraZeneca, Daiichi Sankyo
Used for: HER2-positive breast cancer
Est. 2024 sales: $2.4 billion
After posting impressive tumor-busting results in a pivotal phase 2 trial, AstraZeneca and Daiichi Sankyo’s antibody drug conjugate Enhertu raced to an FDA approval in the final days of 2019. Now, with a launch underway, those impressive results could spell blockbuster sales for the drugmakers.
Enhertu (fam-trastuzumab deruxtecan-nxki) could secure around $2.4 billion in global sales by 2024, according to EvaluatePharma, putting it well on top of the most highly anticipated new drug launches of the year.
AstraZeneca picked up co-marketing rights for the drug in a $6.9 billion deal with Daiichi in March 2019, shelling out a hefty sum for what could prove to be a blockbuster. Jefferies analysts argued at the time of the pickup that the addition would provide a pipeline boost for AstraZeneca with a possibility for expanded indications in the future.
The FDA approved Enhertu in late December as a treatment for patients with inoperable or metastatic HER2-positive breast cancer who have already failed on at least two other treatments in the metastatic setting. AstraZeneca and Daiichi will jointly develop and sell the drug outside of Japan, where Daiichi holds exclusive rights.
Enhertu will likely launch this month at a per-patient cost of around $13,300 per month, SVB Leerink analyst Andrew Berens, M.D., said in a note to investors after the approval. At that price, Enhertu could reach $68 million in sales in 2020, with a peak sales estimate of $2.5 billion, Berens said.
Enhertu is made up of Roche’s Herceptin HER2-targeted antibody and a topoisomerase I inhibitor payload and is being tested in gastric cancer as well as in non-small cell lung and colorectal cancer.
The FDA based its approval on the phase 2 Destiny study, where Enhertu shrank tumors in 61% of patients and eliminated them completely in 6%, Daiichi said. A 97% disease control rate included patients who saw some tumor shrinkage but not enough to be considered responders.
Destiny patients had tried a median of five prior treatments but had essentially run out of options. Patients with HER2-positive breast cancer typically receive Roche’s Herceptin or Perjeta early in their treatment, and when they relapse, they can go on Kadcyla, Genentech’s HER2-targeting antibody drug conjugate.
“Since the beginning of our clinical trial program four years ago, we have focused on the opportunity to transform the treatment landscape for patients with HER2 positive metastatic breast cancer, and we are extremely proud of how quickly we delivered Enhertu to patients in the U.S., as Enhertu represents one of the fastest-developed biologics in oncology,» Antoine Yver, M.D., Daiichi’s global head of oncology R&D, said in a release.
Enhertu will come with a boxed label warning for interstitial lung disease and embryo-fetal toxicity after 9% of Destiny patients developed an ILD during treatment. Nearly all of the Destiny patients (99%) experienced side effects, including nausea, vomiting and low white blood cell counts. Fifteen percent stopped treatment because of side effects, the company said.
Company: Bristol-Myers Squibb
Used for: multiple sclerosis
Est. 2024 sales: $1.598 billion
Ozanimod was a key component of Bristol-Myers Squibb’s $74 billion acquisition of Celgene. In fact, part of the payout to Celgene shareholders depends on FDA approvals for three pipeline drugs—including this multiple sclerosis candidate—by the end of 2020.
It was also partly responsible for Celgene’s sale at a relatively cheap price, after the FDA initially refused to even review the ozanimod application for approval, citing shortcomings in the pharmacology parts of the filing. That refusal-to-file news sent the company’s stock tumbling in early 2018.
Celgene refiled in March 2019, and now, as the new owner, Bristol-Myers is looking at a decision in relapsing forms of MS by March 25, 2020.
Management has high hopes for ozanimod, having pegged its peak sales to the tune of $5 billion. Among analysts polled by EvaluatePharma, the consensus for 2024 sales is more in the $1.6 billion range. But to break the blockbuster barrier at all, it’ll have to compete with several FDA-approved drugs, as well as promising experimental drugs.
Ozanimod is an S1P receptor modulator, designed to dampen inflammatory activity by preventing immune cells from exiting the lymph nodes into the central nervous system. Multiple existing blockbusters and blockbuster hopefuls are targeting the same mechanism. These include Novartis’ $3.3-billion-sales-per-year Gilenya, which is entangled in a patent fight and could face generics sooner than originally expected.
Celgene had touted ozanimod’s better safety profile than the older Novartis medication, particularly with regard to liver toxicities and heart side effects. But the MS up-and-comer has other, newer therapies to worry about.
Novartis also markets Mayzent, which, like ozanimod, selectively binds to S1P subtypes 1 and 5. Despite its own billion-dollar estimate, Mayzent only racked up $4 million in third-quarter sales. During a conference call in October, Novartis executives attributed the slow launch to a 90-day lag in getting patients through the reimbursement process. Such a setback could be a warning for MS greenhorn Bristol-Myers about the tough road ahead.
Also in the competitive oral MS field are Sanofi’s Aubagio, Biogen’s Tecfidera and its newly approved follow-up drug Vumerity—said to boast better gastrointestinal tolerability than its predecessor—and Merck KGaA’s Mavenclad. Besides, Johnson & Johnson’s S1P candidate ponesimod recently showed it could top Aubagio at lowering relapsing MS patients’ annual relapse rate in a phase 3 trial. And Mitsubishi Tanabe is developing another S1P agonist, amiselimod (MT-1303).
Injectables represent a rising force to be reckoned with, most notably Roche’s fast-growing antibody drug Ocrevus. Crowned as the most successful launch in Roche’s history, Ocrevus, administered only twice yearly, generated sales of CHF 2.66 billion in the first nine months of 2019, up 57% year over year.
Then there’s Novartis’ repurposed leukemia drug Arzerra (ofatumumab). Yet unapproved in MS, it cut MS relapse rates by 50.5% and 58.5% respectively in two phase 3 studies when compared with Aubagio. A recent survey by SVB Leerink analysts showed that doctors really like this new Novartis drug. Physicians argue it offers “‘Ocrevus-like’ efficacy, with the convenience of self-administration” once every month, the analysts said.
However, all those drugs face a potentially damaging impact in the form of a patent challenge against market-leading Tecfidera from Mylan. The U.S. Patent and Trademark Office is scheduled to decide by early February whether Biogen’s argument for 2028 exclusivity is valid. If not, a cheaper copycat could enter as soon as this year, threatening not just Biogen’s $4.3 billion haul from the originator in 2018 but also the entire market.
On top of MS, Bristol-Myers is also gunning for ozanimod’s use in inflammatory bowel disease, where it’s in phase 3 testing.
Used for: high LDL cholesterol
Est. 2024 sales: $1.529 billion
Novartis basically paid $9.7 billion for cholesterol-lowering therapy inclisiran when it bought The Medicines Company last year. Is the price too high for a PCSK9-targeting drug, whose class has been suffering a slow launch? The Swiss drugmaker itself seems to think so, but there’s a chance it could prove the idea wrong.
Existing PCSK9 drugs—Amgen’s Repatha and Sanofi and Regeneron’s Praluent—have not lived up to their early hype. After initial reimbursement pushback and a price-cut match-up, the current annualized sales for the two marketed drugs combined are just $1 billion, with Repatha ahead at $168 million in the third quarter.
That’s four years into their launches—and the Street had originally expected multibillion-dollar sales for each. In fact, the market is so underperforming that Sanofi has lost confidence and rejigged its Regeneron partnership by returning U.S. Praluent rights.
Inclisiran has put up comparable data on its ability to lower bad cholesterol. The new drug is different from the two antibodies in that it uses a small-interfering RNA to target PCSK9. It’s also given twice a year after two lead-in doses, whereas the two FDA-approved drugs are given every two weeks or once a month.
But Wolfe Research analyst Tim Anderson, M.D., previously argued that a convenience edge won’t be enough to turn the tide, suggesting “the commercial case—injectable drugs for a ‘silent’ disease, entering a highly-genericized category—would be challenging.”
It all comes down to execution, analysts from Evercore ISI and Jefferies said. But there’s a high bar to clear: Evercore ISI’s Umer Raffat figures inclisiran needs to hit more than $2 billion in peak annual sales to justify the $9.7 billion purchase price. EvaluatePharma researchers say the drug could reach $1.529 billion in 2024.
One key piece to inclisiran’s launch puzzle will be cardiovascular outcomes data, which both Repatha and Praluent have. Although the new medication won’t have that data till 2024, when the Orion-4 trial is expected to deliver outcomes results, MedCo management has argued that LDL-c is already a validated marker to predict CV benefits—but it remains to be seen whether payers think the same.
In terms of marketing, Novartis is obviously a better owner for inclisiran than MedCo, what with the Big Pharma’s decadeslong CV experience with its valsartan franchise, including the fast-growing combination drug Entresto. Despite bearing different indications, Entresto prescribers amount to about 80% of prescribers of statins or existing PCSK9s in the U.S., Novartis CEO Vas Narasimhan, M.D., has said. That means the company only needs “an incremental few hundred reps” to field inclisiran. As for the ex-U.S. market, the existing team could cover about 90% of doctors, he said.
To kick things off, at the J.P. Morgan Healthcare Conference in January, Narasimhan and Britain’s National Health Service unveiled a “population-level agreement” that would make inclisiran available for high-risk cardiovascular disease patients upon its expected regulatory approval. Under the agreement, the drug will also be tested in a large-scale U.K. clinical trial in primary prevention, in which heart disease has yet to develop in at-risk patients.
In the U.S., MedCo—now Novartis—submitted the new drug application for inclisiran to the FDA in December 2019.
Drug: Evrenzo (roxadustat)
Companies: FibroGen, AstraZeneca and Astellas
Used for: anemia associated with kidney disease
Est. 2024 sales: $1.488 billion
FibroGen’s anemia drug Evrenzo (roxadustat) has already won approvals and national coverage for both nondialysis-dependent and dialysis-dependent kidney patients in China, where it shares rights with AstraZeneca. In Japan, its partner Astellas has nabbed a green light for dialysis patients.
In the U.S., FibroGen filed for approval in both indications in December 2019. EvaluatePharma predicted the drug could turn up $1.488 billion by 2024 sales. Another team of analysts is a lot more bullish, though; SVB Leerink figures China alone could deliver $1.4 billion in 2025, and that its global number could range between $3 billion and $3.5 billion by that time.
Roxadustat is the first in the hypoxia-inducible factor prolyl hydroxylase inhibitor class to be approved anywhere. Different from Amgen and Johnson & Johnson’s standard-of-care injection Epogen/Procrit, oral roxa corrects anemia by mimicking a response to reduced oxygen levels.
Pooled phase 3 analyses showed roxa triggered a significantly higher increase in hemoglobin levels in dialysis-dependent patients compared with Epogen/Procrit, with mean change from baseline averaged over weeks 28 to 52 standing at 1.22 g/dL and 0.99 g/dL respectively. In nondialysis-dependent patients, the numbers were 1.85 g/dL for roxa and 0.13 g/dL with placebo. For men, normal hemoglobin levels range from 14.0 g/dL to 17.5 g/dL; for women, it’s between 12.3 g/dL and 15.3 g/dL, according to the Cleveland Clinic, though the standards vary slightly among institutions.
Investigators pitted roxa against placebo in nondialysis patients because traditional erythropoietin-stimulating agent therapy isn’t approved for that group; the cardiovascular risks are too great. Roxa hopes it could make a difference for these patients—and access to that market is a much bigger opportunity than sticking to dialysis patients alone. According to the CDC‘s most recent data, about 4 million people in the U.S. know they have chronic kidney disease in 2019. In contrast, according to U.S. Renal Data System data cited by AZ, about 509,000 U.S. patients were receiving dialysis in 2016.
In the pooled analysis, the risk of major adverse CV events observed in the roxa patients was comparable to that in the placebo arm. However, a hazard ratio of 1.08 means roxa’s rate was slightly higher, though not statistically significant.
That trend worried some investors, who feared the FDA might stonewall roxa in the larger nondialysis indication. FibroGen says the idea is erroneous.
Enrique Conterno, FibroGen’s newly installed CEO and a 27-year Eli Lilly veteran who previously headed the Indianapolis pharma’s diabetes operations, recently told SVB Leerink analyst Geoffrey Porges that many diabetes drugs had won CV label expansions with much higher hazard ratios, all the way up to 1.8.
Investors have another worry, too: Because of the Centers for Medicare & Medicaid Services’ “bundled” payment system for U.S. dialysis treatments and services, they’re concerned that CMS might be reluctant to reimburse a new drug outside that package. But FibroGen management pointed Porges to a relatively new rule called the Transitional Drug Add-on Payment Adjustment—which allows additional payments for new therapies—suggesting the company will apply for coverage through that path.
Roxa could face some in-class competition soon. These include GlaxoSmithKline’s daprodustat, filed for approval in Japan under a collaboration with Kyowa Kirin. And Akebia Therapeutics and local partner Mitsubishi filed their rival, vadadustat, in Japan in July 2019. In the U.S., Akebia has teamed up with Japanese pharma Otsuka on the anemia treatment.
5. Sacituzumab govitecan
Drug: sacituzumab govitecan
Used for: triple-negative breast cancer
Est. 2024 sales: $1.44 billion
Triple-negative breast cancer (TNBC) therapy sacituzumab govitecan didn’t exactly sail through the FDA: A complete response letter (CRL) in early 2019 nearly sank its approval hopes. But drugmaker Immunomedics is taking a new tack with regulators––and it could help the drugmaker reach blockbuster sales.
Sacituzumab govitecan could reach $1.44 billion in 2024 global sales, according to EvaluatePharma, as a third-line treatment for late-stage, metastatic TNBC.
Immunomedics resubmitted its application for the drug in early December, and the FDA has a decision date set for June, the company said.
Sacituzumab govitecan is an antibody drug conjugate including the active ingredient in chemotherapy Onivyde. The chemotherapeutic treatment will be tasked with stacking up against Roche I-O therapy Tecentriq, which was approved to treat TNBC in March.
In a note to investors earlier this month, Cantor analyst Varun Kumar, Ph.D., posited that sacituzumab could be a strong candidate for an acquisition if it nabs an FDA approval as a third-line-or-later treatment. Kumar put the drug’s possible acquisition value at $7.1 billion.
Immunomedics is hoping for a better second time around after the FDA slapped sacituzumab govitecan with that CRL. Manufacturing issues triggered the rejection, the company said. The agency didn’t ask for new clinical or preclinical data, though, which would have delayed the application further.The CRL was only the start of Immunomedics’ problems, though. When pressed by analysts after the CRL was made public, company executives dodged questions about whether the rejection stemmed from a preapproval inspection of its plant last year. The FDA was more forthcoming; it released a Form 483 from the inspection showing big issues with data integrity at the Morris Plains, New Jersey, plant where sacituzumab govitecan would be made.
A management shakeup ensued. CEO Michael Pehl hit the exit in February 2019, swiftly followed by R&D lead Robert Iannone, M.D., who later joined Jazz Pharmaceuticals. According to Immunomedics’ website, both positions remain unfilled.
But Immunomedics said it has resolved its manufacturing problems and felt comfortable bringing sacituzumab govitecan back up for regulatory scrutiny.
Company: Gilead Sciences
Used for: rheumatoid arthritis
Est. 2024 sales: $1.28 billion
Gilead Sciences, with its hepatitis C glory days behind it, is working to push into the anti-inflammatory market. And with an FDA filing now under speedy review, rheumatoid arthritis candidate filgotinib could go a long way to helping Gilead toward that goal.
While the FDA hasn’t listed a decision date, filgotinib is already under review at the agency and could rake in global sales of $1.28 billion in 2024, according to EvaluatePharma. The FDA accepted Gilead’s application in late December with a priority review voucher attached, likely spelling a speedy review and decision from the administration.
At the J.P. Morgan Healthcare Conference in January, Gilead commercial chief Johanna Mercier focused on the strength of filgotinib’s clinical data and said physician feedback has so far been very positive. The company foresees a total of five indications for filgotinib launching in the next four years, so it’s keeping not only the short term but also the medium-to-long term in mind, she said.
Filgotinib has already been filed for approval in Europe and Japan, but the U.S. market is where the big bucks will come from, making this an important milestone for Gilead. The Big Biotech has long had a stake in filgotinib, with recent data readouts showing the medication is better than methotrexate alone while suggesting it has an edge over AbbVie’s Humira, the aging blockbuster rheumatoid arthritis incumbent.
Results shared to date suggest Gilead has enough positive evidence to get filgotinib approved. The harder question is whether the evidence is strong enough to get filgotinib a cleaner label than its rivals’ or otherwise give it an advantage that translates into the hoped-for blockbuster sales.
The safety of filgotinib is a particular source of ongoing uncertainty. Gilead and Galapagos think filgotinib is free from some of the safety problems that blighted Pfizer’s Xeljanz and Eli Lilly’s Olumiant, along with other members of the JAK family. However, AbbVie’s Rinvoq, which, like filgotinib, is specific to JAK1, received a black box warning when it won FDA approval earlier this year.
7. Palforzia (AR101)
Drug: Palforzia (AR101)
Used for: peanut allergy
Est. 2024 sales: $1.28 billion
Aimmune is taking a big swing with its homeopathic medicine Palforzia (AR101) in a food allergy field where avoidance is the norm. Despite skepticism from some market watchers, the FDA’s approval could put Palforzia on the path to blockbuster status in the next few years.
Palforzia could hit an estimated $1.28 billion in sales by 2024, according to EvaluatePharma, as a peanut allergy immunotherapy that works by desensitizing patients through gradual doses of peanut protein.
On Friday, the FDA approved Palforzia as a treatment for peanut allergy reactions, including anaphylaxis. In September, an FDA advisory committee panel voted 7-2 to recommend approval of the drug.
In a note to investors in March 2019, Credit Suisse analyst Vamil Divan, M.D., said physicians were largely positive on the clinical need for Palforzia but raised concerns about the drug’s commercial chances.
«We believe there are more investor questions on the commercial opportunity for the product given the need for frequent office visits and GI side effects during the up-dosing period,» Divan wrote. «However, we see significant interest in the product from physicians and patients/parents, a side effect profile that appears manageable and a commercial opportunity that can be addressed through a targeted sales force.»
In its recommendation, the FDA’s advisory committee voted 8-1 in favor of a safety plan called a Risk Evaluation and Mitigation Strategy, or REMS, proposed by the agency, to support the use of Palforzia in children aged 4 to 17.
This plan mandated that any patient potentially prescribed Palforzia has a valid prescription for injectable epinephrine, caregivers/patients attest to carrying injectable epinephrine while taking Palforzia and the initial dose escalation and the first dose of each up-dosing level must be given at a certified site capable of treating systemic allergic reactions.
The FDA based its review on Palforzia’s yearlong Palisade trial, which tested the treatment against placebo in 555 patients aged 4 to 55. The vast majority of them were children and adolescents, the age group for which Aimmune sought approval.
At the start of the trial, patients could not tolerate 100 mg or less of peanut protein—a fraction of a single peanut, which weighs in at about 300 mg. Patients were given a higher dose of the treatment every two weeks until they reached a maintenance dose of 300 mg. After one year of treatment, 67% of patients in the intent-to-treat population could tolerate 600 mg of peanut protein, about two peanuts or a small bite of a peanut butter sandwich, compared to 4% of patients who received placebo.
In early 2019, a government shutdown delayed the FDA’s review of Palforzia as a direct consequence of a “lapse in appropriations.» In other words, the gap hampered the agency’s ability to raise further funding through user fees, the drugmaker said.
8. Valoctocogene roxaparvovec
Drug: valoctocogene roxaparvovec
Company: BioMarin Pharmaceutical
Used for: hemophilia A
Est. 2024 sales: $1.212 billion
While Roche struggled with its $4.3 billion takeover of Spark Therapeutics thanks to unexpected antitrust concern over the latter’s hemophilia gene therapy pipeline, a competitor has advanced with regulatory filings.
BioMarin Pharmaceutical submitted the application for its rival hemophilia A gene therapy, valoctocogene roxaparvovec, to the FDA at the end of 2019, just as the EMA accepted its package for accelerated review.
Industry watchers were confused by the U.S. Federal Trade Commission’s extra scrutiny around Roche-Spark because its SPK-8011 was not even the furthest hemophilia A gene therapy program in development—or the best, by the first look of it.
Three-year phase 2 data for BioMarin’s valrox showed that the one-time therapy maintained a median annual bleed rate of 0, or 0.7 on average, which translates into a reduction of 96% from patients’ study entry. Annual factor VIII usage also dropped to a median of 0 or an average of 5.5, versus 138.5 and 136.7, respectively, before treatment.
As for Spark’s SPK-8011, the drug led to a 94% reduction in annual bleeds among 12 phase 1/2 participants four weeks after infusion. But in August 2018, Spark’s stock slid at the news of some patients having experienced an immune response-related sharp decline in factor VIII levels. That slide basically opened the buyout window for Roche.
In May 2019, BioMarin unveiled that its phase 3 study also met the prespecified criteria for regulatory submissions. Now, as BioMarin’s applications are on EU and U.S. regulators’ desks, Roche/Spark is still in a lead-in study that tests traditional factor VIII replacement therapy for a future SPK-8011 phase 3.
Expecting a possible launch in 2020, BioMarin has stepped up its manufacturing efficiency, having increased the capacity for its gene therapy facility in Novato, California, from about 4,000 doses a year to up to 10,000 per year.
The upgrade is important “because first-mover advantage in gene therapy is fundamental in a sense that every time you treat a patient that patient is off the market,” BioMarin Chairman and CEO Jean-Jacques Bienaime said at the annual J.P. Morgan Healthcare Conference.
Being able to produce 10,000 doses means the company can treat the entire existing severe hemophilia A U.S. patient population in two years—not that it will—ahead of a potential approval for SPK-8011, Bienaime explained.
Once approved, valrox will first compete for noninhibitor patients against Roche’s antibody drug Hemlibra, which has been rapidly stealing share from factor VIII treatments and contributed CHF 921 million ($950 million) to Roche’s top line in the first 9 months of 2019.
Citing a recent Citi survey of 60 U.S. hematologists, Bienaime showed that these physicians expect to switch 29% of Hemlibra patients and 34% of factor VIII patients over to gene therapy within a year.
EvaluatePharma predicts valrox could reach $1.212 billion in annual sales by 2024.
Company: Biohaven Pharmaceutical
Used for: migraine
Est. 2024 sales: $897 million
Three marketed CGRP drugs—Amgen and Novartis’ Aimovig, Teva’s Ajovy and Eli Lilly’s Emgality—are injections approved to prevent migraine. Biohaven Pharmaceutical is aiming to bring an oral option to the table.
But first, instead of the prevention market, Biohaven is gunning for an approval of oral rimegepant in the acute treatment setting. It’s used a priority review voucher for an expedited six-month review, with an FDA decision now expected by the end of June.
According to topline results from a phase 3 trial, about 19.6% of rimegepant patients were completely free from pain two hours after taking the tablet, versus 12.0% in the placebo arm.
In a separate phase 3, an orally dissolving formulation demonstrated earlier onset of action, as the pain relief benefit of rimegepant started to numerically separate from placebo as early as 15 minutes after dosing, and the difference turned statistically significant at one hour. After two hours, 21.2% of rimegepant and 10.9% of placebo patients were pain-free.
Although Biohaven has already lost the first-to-market oral CGRP race to Allergan, which nabbed an FDA nod for Ubrelvy (ubrogepant) at the end of 2019, industry watchers figured rimegepant represents a more successful sales opportunity. EvaluatePharma analysts previously predicted the Allergan version would generate $302 million by 2024, well below rimegepant’s projected $897 million.
There’s also Lilly’s newly launched oral drug Reyvow, the first in the serotonin (5-HT)1F receptor agonist class. But SVB Leerink analyst Marc Goodman suggested that the therapy’s side effects, including dizziness and drowsiness, could limit its sales performance to under $500 million at peak.
The question is, can small Biohaven, with rimegepant as its first commercial product, challenge all those Big Pharma players? Biohaven CEO Vlad Coric, M.D., has expressed confidence that it can. The company “will strive to outpace them with a more efficient and innovative commercial launch, using modern-day strategies,” he told FiercePharma in a recent email interview.
While acute treatment could be rimegepant’s first indication, Biohaven is also looking to expand into the larger prevention market. It finished enrollment of more than 1,600 patients in a phase 3 trial, though it hasn’t yet released topline data as originally planned for the fourth quarter of 2019.
To further complicate the market landscape, neurology specialist Lundbeck recently paid $2 billion to acquire Alder BioPharmaceuticals and its late-stage CGRP quarterly infusion drug eptinezumab, which expects an FDA ruling in migraine prevention by Feb. 21, 2020. RBC Capital analyst Brian Abrahams previously pegged that drug’s peak sales at $750 million.
Used for: spinal muscular atrophy
Est. 2024 sales: $803 million
In the extremely pricey spinal muscular atrophy (SMA) market, Novartis and Biogen have taken the lead with their drugs passing FDA muster in 2019. But Roche and partner PTC Therapeutics are aiming to take their own late-stage SMA candidate to market, and a priority review could help get it in physicians’ hands faster.
Roche’s risdiplam could hit $803 million in global sales by 2024, according to EvaluatePharma, if the FDA gives it the green light by its May decision deadline.
The FDA agreed to review the drug in November 2019 based on 12-month data from a study called Firefish in type 1 SMA patients, plus data in type 2 and 3 patients from a study called Sunfish. The drugmaker is testing the candidate in a broad patient population, ranging from newborns to age 60, including those who have taken other SMA medications.
In mid-January, risdiplam hit its primary endpoint in another phase 3 trial in infant patients with type 1 SMA, which assessed whether infants were able to sit without support for at least five seconds after receiving daily doses of risdiplam for one year.
Risdiplam, a survival motor neuron 2 splicing modifier, is an orally administered liquid that patients would take at home. Biogen’s Spinraza is an injection with several loading doses over the first two-and-a-half months and maintenance doses every four months thereafter. Novartis’ gene therapy Zolgensma is a one-time treatment for patients under 2.
If the oral drug reaches approval, Cantor analyst Alethia Young said in a recent note that it could be a «game changer» in SMA.
Analysts believe the new Roche drug could bring in $2.5 billion to $3 billion at peak. As of November 2019, Biogen’s Spinraza had pulled in $1.55 billion. Zolgensma had generated $160 million since launch.
Risdiplam scored the FDA’s orphan drug designation in January 2017 and fast-track designation in April 2017. While the FDA’s official decision date is May 24, Cantor’s Young wrote that an approval could come earlier than that.
Regardless of when the drug scores a nod, analysts expect risdiplam to bring big changes to the market upon launch. Young wrote that it’ll “meaningfully change the SMA landscape in 2020.»
Biogen launched its Spinraza first in the field and the rollout has surpassed all expectations. Novartis has hit some hiccups with Zolgensma, including a data manipulation controversy that didn’t dent demand. Analysts still believe the drug will generate blockbuster sales.