As the world haphazardly endeavors to defeat a deadly virus, the pharma industry has found itself charged with delivering a miracle cure — in a compressed, unprecedented time frame, no less. But as drug developers swim hard and fast to neutralize COVID-19 with vaccines and therapeutics, they are also working to get other important drugs approved amid looming threats of halts and delays.
Indeed, even as the industry prioritizes everything “essential” as the pandemic rages, could the current emphasis on speed and efficiency spur a new (and faster) normal for drug development timelines, approvals and regulations? Time will tell, but 2021 is looking to be a big year for the pharma pipeline.
COVID-19 vaccines have been in the spotlight this year, a focus which is only expected to intensify in 2021. While usual vaccine development and approvals take decades, multiple drug developers are aiming to shorten this process to around 18 months. But while Pfizer, Moderna and Johnson & Johnson are among the current frontrunners with novel vaccine approaches, the lack of precedence for both mRNA and adenoviral vector vaccines render them high-risk, high-reward endeavors. With emergency use authorizations (EUAs) expected by year end or early 2021, we should be hearing major Phase III data verdicts for these vaccines around mid-2021.
Meanwhile, significant progress has been made with COVID-19 therapeutics, particularly in the wake of Gilead’s Veklury (remdesivir) approval in October. While analysts anticipate the drug to bring in around $1.5 billion by the end of 2020, new mixed data and a high price has drawn some skepticism on whether Gilead can sustain its market traction. This may pave the way for other candidates, such as Regeneron’s antibody cocktail REGN-COV2 or InflaRx’s C5a inhibitor IFX-1.
Beyond COVID-19, many promising drugs continue to tick along in late-stage trials and approval discussions. Oncology, ever an essential priority for drug developers, boasts a robust pipeline, led by J&J/Legend Biotech’s CAR-T therapy Cilta-cel. It’s expected to be approved next year for multiple myeloma, along with Bristol Myers Squibb’s direct competitor Ide-cel.
Next year could also be a pivotal year for Iovance, which is set to release data and file for the approval of Lifileucel, potentially the first cell therapy approved for solid tumors. New combination therapies could also offer incremental upside to the current treatment paradigm, with Nektar/Bristol Myers Squibb’s bempegaldesleukin/nivolumab combo vying for approval to treat melanoma and other solid tumors.
Meanwhile in neurology, a final verdict on Biogen’s controversial resurrection of aducanumab for Alzheimer’s disease — a potential $15 billion dollar drug — is expected in March 2021. However, a negative vote in early November by the FDA advisory committee doesn’t bode well for its chances. Though a future approval hasn’t been ruled out, more studies could be a likely scenario. Meanwhile, AbbVie and Biohaven Pharmaceuticals continue to evolve their migraine franchises with 2021 regulatory filings for atogepant and zavegepant, respectively.
Non-COVID immunology drug development continues to be dominated by skin conditions, with UCB’s bimekizumab on the cusp of approval for moderate-to-severe psoriasis patients. Similarly, BMS is awaiting additional Phase III data for its oral psoriasis drug BMS-986165, which could be a game-changer, in terms of convenience, for patients with moderate-to-severe disease.
Several other candidates are expected to be approved for atopic dermatitis. The wild card might be Arena’s etrasimod, currently in Phase III trials for ulcerative colitis, which has potential in other autoimmune indications.
Gene therapy continues to make its mark in blood disorders, with both UniQure and Biomarin developing hemophilia candidates dangling the promise of one-time therapies for patients who are used to aggressive treatment regimens. Meanwhile, Bluebird Bio’s LentiGlobin awaits further approvals for sickle cell disease and beta thalassemia next year.
Elsewhere, Myovant is attempting to battle AbbVie in the women’s health space, via an expected approval of Relugolix for uterine fibroids. Merck looks forward to a long-awaited approval verdict for its heart failure drug vericiguat in a niche population. And Fabry’s patients could have a new and safer enzyme replacement therapy option with the impending approval of Protalix’s PRX-102.
In other words, buckle up in advance of another epic and unpredictable year for pharma.
(Agents profiled in this report were chosen in consultation with inThought Research, inDemic, Adis R&D Insight, Ipsos Healthcare and GlobalData. Analyses of featured products include the latest clinical data, revenue forecasts, expected launch dates and likelihood of success as of early November 2020.)
COVID-19
Most anticipated products
COVID-19 Vaccines
BNT162b2
Pfizer/BioNtech, Phase III
Type: mRNA vaccine
Where the clinical trials are: BNT162b2 is an mRNA vaccine, which encodes an optimized SARS-CoV-2 full-length spike glycoprotein at a 30µg dose level in a two-dose regimen. Interim data as of early November showed that the vaccine was 95% effective. While emergency use authorization could arrive as early as late-Q4 2020, the primary analysis of the Phase III trial is expected in Q2 2021, which could enable mass vaccinations thereafter.
InDemic comment: Pfizer’s criteria for what constitutes a “case” of COVID-19 is less stringent than that of its competitors, and this will allow the company to tally up cases more quickly for an earlier readout. While it could generate some skepticism from regulators and the scientific community, Pfizer is also tracking “severe cases” of COVID-19 and other metrics for efficacy in its vaccine trial. Pfizer’s refusal to accept federal money for its vaccine development shows confidence in its technology. — Dr. Sam Sun, director, InDemic Foundation
What physicians are saying: This candidate was poised to apply for emergency use authorization by late November. Orders for millions of doses have already been placed by U.S., EU and Japanese governments, highlighting the readiness to react to the imminent availability of this much-needed product. — Rhoda Schmuecking, head, virology and vaccines CoE, Ipsos
mRNA-1273
Moderna, Phase III
Type: mRNA vaccine
Where the clinical trials are: mRNA-1273 is an mRNA vaccine that encodes a SARS-CoV-2 spike protein that is stabilized in the prefusion (before infection) conformation. The vaccine is in an ongoing Phase III trial with early reports showing a 94.5% efficacy rate and, even if interim analyses spur emergency use authorization for high-risk individuals in late Q4 2020 or early Q1 2021, a full analysis for widespread approval is due in Q2 2021.
InDemic comment: Moderna is a pioneer in developing mRNA therapeutics. Overall, Moderna has shown consistency and transparency with its vaccine program, which may aid in its uptake and eventual rollout to the public. Its strict definition of what constitutes a COVID-19 case could mean it takes longer for Moderna to produce a final readout. However, that sacrifice could generate greater significance for its readout. — Sun
What physicians are saying: With the first interim analysis in November, the possibility of vaccines from Pfizer/BioNTech and Moderna being made available for emergency use authorization before the end of this year certainly makes this a very dynamic space to watch. — Schmuecking
Ad26.COV2.S
Johnson & Johnson, Phase III
Type: human adenoviral vector
Where the clinical trials are: Ad26.COV2.S is composed of a recombinant, replication-incompetent adenovirus type 26 (Ad26) vector, constructed to encode the SARS-CoV-2 spike protein. J&J’s 60,000-patient Phase III trial is testing the single-dose vaccine versus placebo. However, the trial was paused in mid-October due to a safety signal of unknown significance. While this could impact its future in the race, the trial was resumed in late October.
InDemic comment: The vaccine’s one-dose regimen makes it attractive compared to other candidates. Its recent safety event may not be as serious as the rare neurological reactions experienced by two patients on AstraZeneca/Oxford University’s adenoviral vaccine trial, which was paused in September. J&J’s vaccine technology, Advac, has been used in other approved vaccines, and 100,000 patients have been vaccinated with this technology without a concern. — Sun
What physicians are saying: Should the late-stage study prove positive, J&J expects to be able to supply 1 billion doses of its vaccine beginning early next year. While this is later than the mRNA vaccine candidates, J&J’s boldness with the size of trial and one-shot dose makes it a very interesting option added to the mix. — Schmuecking
COVID-19 Therapeutics
Veklury (remdesivir)
Gilead, post-approval
Type: anti-viral
What the clinical trials have shown: Remdesivir (brand name Veklury) received full approval from the FDA in October, making it the first antiviral available to treat COVID-19. That approval was based on data from the ACTT-1 study sponsored by the National Institutes of Health, which found a significant benefit in time to recovery and a non-significant trend toward improved survival at 29 days. However, preliminary findings from the WHO Solidarity trial, which followed 11,366 adults at 405 hospitals in 30 countries, found that remdesivir has little or no impact on survival.
InDemic comment: The available data indicate that remdesivir does not improve mortality – and might even worsen mortality – in COVID-19 patients with severe disease who require high levels of supplemental oxygen or mechanical ventilation. For that reason, it may only be appropriate to consider this drug for hospitalized COVID-19 patients with mild to moderate disease. The conflicting data on survival may also impact Gilead’s ability to command the high price of $2,600 per course. — Sun
What physicians are saying: In lieu of an available vaccine for COVID-19, remedisvir, along with other antivirals in development, provides a valuable option in physicians’ treatment armamentarium. — Schmuecking
REGN-COV2 (antibody cocktail)
Regeneron, Phase III
Type: passive immunotherapy
What the clinical trials have shown: Results from Regeneron’s ongoing Phase II/III trial in non-hospitalized patients showed that its antibody cocktail significantly reduced viral load and patient medical visits. The company will use this data for an emergency use authorization of REGN-COV2 low-dose to treat mild-moderate patients, for potential 2021 approval. However, the drug’s potential is less clear in severe patients: In late October, an independent monitoring committee recommended halting enrollment of patients with high oxygen requirements on its hospitalized patient trial, due to an unfavorable risk/benefit profile.
InDemic comment: REGN-COV2 and other antibody therapies against COVID-19 are expected to show substantial efficacy in reducing hospitalizations. REGN-COV2 also fills an unmet need in that it can be used for outpatients with COVID-19, whereas both remdesivir and dexamethasone are used for hospitalized patients. — Sun
What physicians are saying: A premature EUA could hamper ongoing investigations needed to solidify efficacy data and offer better utility insight, according to physicians interviewed by Manasi Vaidya and Reynald Castaneda, associate editors of GlobalData’s Pharma investigative news team. Larger datasets on clinically relevant secondary endpoints, such as reduced hospitalization, are needed for stronger EUA consideration. The ability to manufacture adequate mAbs to provide access to patients is likely to be an issue in the event of a premature EUA, the experts added.
IFX-1
InflaRx, Phase III
Type: complement inhibitor
What clinical trials have shown: In previous trials, IFX-1 was shown to reduce kidney injury, lung emboli and mortality from multi-organ failure, so IFX-1’s Phase III trial was initiated to enroll severe COVID-19 patients, with a primary endpoint of mortality. Interim data is expected in March 2021, dependent on enrollment.
InThought comment: The Phase III trial of IFX-1 is enrolling a specific population of very sick COVID-19 patients. Its primary endpoint is objective and clinically meaningful, and its Phase II trial showed that IFX-1 may reduce mortality of COVID-19 multi-organ failure. With a dearth of treatments for severe/critically ill COVID-19 patients, we believe IFX-1’s interim data in early 2021 will support the use of complement inhibitors for severe COVID-19 pneumonia. It could also be synergistic with the use of dexamethasone. — Sun
Oncology
Most anticipated products
Cilta-cel (JNJ4528 or LCAR-B38M)
Johnson & Johnson, pre-registration
Indication: late-stage multiple myeloma
What the clinical trials have found: Cilta-cel is a CAR-T therapy targeting BCMA (B-cell maturation antigen) and a Phase Ib/II study in refractory multiple myeloma is expected to yield a 2021 approval. Longer-term follow-up data from the study demonstrated a 100% overall response rate and an 86% stringent complete response rate at a median of 11.5 months.
InThought comment: The 100% response rate is the main focus of this drug, which, along with its FDA breakthrough therapy designation, has placed Cilta-cel as an emerging frontrunner in the competitive BCMA space. — Amanda Weyerbacher, senior principal, inThought Research
Credit Suisse revenue forecast: $427 million by 2025
What physicians are saying: Despite its race (with Bristol Myers Squibb’s Ide-cel) to be the first CAR-T therapy approved for multiple myeloma, there is likely to be high anticipation for Cilta-cel as physicians and patients (who have progressed through a number of drug classes) look for strong and durable outcomes — Amy Butcher, director, global oncology monitor, Ipsos
Mosunetuzumab
Roche, Phase I/IIb
Indication: relapsed or refractory follicular lymphoma
What the clinical trials have found: Mosunetuzumab is a bispecific T-cell engager that targets CD3 and CD20. The FDA granted breakthrough therapy designation for relapsed or refractory follicular lymphoma in July based on a Phase I/Ib GO29781 study demonstrating high response rates and durable complete remissions in people with relapsed or refractory non-Hodgkin’s lymphoma.
InThought comment: Roche is in active discussions with the FDA for accelerated approval in this indication. Continued updates are expected to build on already promising data, while a subcutaneous formulation could offer at-home administration and patient convenience, which has been set at a higher bar in the time of COVID. — Weyerbacher
Credit Suisse revenue forecast: $250 million by 2025
What physicians are saying: Trial results garnered much attention because it achieved efficacy in patients who had already been through four to five different treatments, and even included patients who had undergone CAR-T therapy. This will be a welcome new weapon for patients with an extremely poor prognosis. — Alessandra Franceschetti, director, global oncology monitor, Ipsos
Bempegaldesleukin/nivolumab
Nektar, Bristol Myers Squibb, pre-registration
Indication: melanoma, RCC, NSCLC
What the clinical trials have found: Nektar and BMS are examining the drug combination for a range of solid tumors, including first-line melanoma, renal cell cancer and lung cancer. Phase II melanoma data showed an overall response rate of 53%.
InThought comment: This combination has shown promising activity across a number of solid tumor indications that improve upon current immunotherapy approaches. While melanoma is the most advanced, renal cancer is looking to come out on top. — Matt Presby, senior analyst, InThought Research
Credit Suisse revenue forecast: $1.75 billion by 2025 in all indications
What physicians are saying: Although early-stage, the data indicate that the combination could lead to durable responses with a possible advantageous toxicity profile over some other immuno-oncology combinations. — Eric Blouin, SVP, Ipsos
Lifileucel (LN-144)
Iovance, Phase II
Indication: metastatic melanoma
What the clinical trials have found: Lifileucel is an autologous cell therapy technology, which uses tumor-infiltrating lymphocytes (TILs) from a patient’s own tumors. In a Phase II metastatic melanoma trial of 66 heavily treated patients, the objective response rate (ORR) was 36%.
InThought comment: Lifileucel could be on track to be the first cell therapy approved for solid tumors. The TILs technology appears to achieve responses on par with or better than currently approved options for second- and third-line melanoma, while responses in cervical cancer (LN-145) are three times greater than current immuno-oncology agents. — Presby
What physicians are saying: While Phase II data has shown good antitumor activity and durable response rates, the drug carries a complex manufacturing and delivery process. A Biologics License Application (BLA) submission is expected in 2021 following the FDA’s request for additional data on the proposed potency assays. — Mo Muhsin, director, oncology, Ipsos
Neurology
Most anticipated products
Aducanumab
Biogen, pre-registration
Indication: Alzheimer’s disease
What the clinical trials have found: Aducanumab is a monoclonal antibody targeting aggregated forms of beta-amyloid found in the brain. Phase III development of the drug started in late 2016 and was stopped in March 2019 after an independent group analysis deemed the trials not worth continuing because the drug was not expected to reach the trials’ primary endpoint.
However, in October 2019, Biogen decided it would submit the data for regulatory approval after re-analyzing a larger dataset that showed aducanumab reduced clinical decline in patients at higher doses. On November 6, the FDA advisory committee verdict voted strongly against approving aducanumab on its current dataset. A final verdict from the FDA is due on March 7.
InThought Comment: It will be more difficult, although not impossible, for the agency to ignore this strong no-confidence stance. Standards need to be kept as high as established. For this population, at least in the beginning, the effects of a pharmaceutical intervention proven efficacious in trials is highly likely to have almost imperceptible clinical effectiveness in real life in many cases, if not the majority. That will be difficult enough. We really need to be as certain as humanly possible here. — Leon Henderson-MacLennan, medical adviser, InThought Research
Credit Suisse revenue forecast: $15.4 billion by 2025
What physicians are saying: There are currently no disease-modifying treatments available for Alzheimer’s. If aducanumab gains approval by the FDA, it could represent the first real signs of hope for the millions of people impacted by this devastating disease. — Scott Morano, VP, healthcare, Ipsos
Atogepant
AbbVie, pre-registration
Indication: migraine
What the clinical trials have found: AbbVie’s atogepant is an oral calcitonin gene-related peptide (CGRP) receptor antagonist being developed for first-line migraine prevention. A positive Phase III trial showed a significant reduction in mean monthly migraine days over 12 weeks compared to placebo. AbbVie is planning regulatory filings in the U.S. and Europe in 2021.
InThought comment: Atogepant has a good shot to ride on the coattails of the rapid adoption of oral migraine and carve out a significant portion of the multibillion-dollar migraine market. Yet more mature data is awaited to elucidate its exact differentiation and positioning to competitors and which patient setting this drug will truly be used for. — Henderson-MacLennan
Credit Suisse revenue forecast: $723 million by 2025
What physicians are saying: The past two years have seen radical improvements in the treatment options available for the prevention of migraine. Atogepant represents another giant step forward with its oral formulation that could make the CGRP receptor agonist much more palatable for PCPs — and therefore more accessible to patients. — Morano
Zavegepant (formerly Vazegepant)
Biohaven Pharmaceuticals, pre-registration
Indication: migraine
What the clinical trials have found: A pivotal Phase II/III study showed that zavegepant at 10mg and 20mg doses was statistically superior to placebo on co-primary endpoints of pain freedom and freedom from most bothersome symptoms at two hours. It also showed rapid onset of pain relief at 15 minutes with a favorable toxicity profile and no signal of hepatotoxicity. An NDA filing is expected in the second half of 2021.
InThought Comment: While more data is still awaited, zavegepant is being watched keenly because of its differentiated intranasal route of administration. This is likely a safe choice and could allow Biohaven to carve out a niche portion of the migraine market. — Henderson-MacLennan
GlobalData consensus forecast: $563 million in 2026
What physicians are saying: CGRP treatments have proven effective for migraine. Zavegepant represents another innovation in not only providing potentially both acute and preventive treatment, but also in an administration-friendly intranasal formulation. It’s also potentially a treatment for COVID-19. — Morano
Immunology
Most anticipated products
Bimekizumab
UCB, pre-registration
Indication: moderate to severe psoriasis
What the clinical trials have found: Bimekizumab is an IL-17 inhibitor that suppresses both IL-17A and IL-17F, which are two key cytokines driving inflammatory processes. UCB has completed three successful Phase III trials of bimekizumab, showing superior skin clearance at week 16 compared to placebo and AbbVie’s blockbuster Humira. The drug has a PDUFA approval date in March 2021 and is also being examined for treatment of psoriatic arthritis and ankylosing spondylitis.
InThought comment: Bimekizumab has shown stronger efficacy compared to IL-17a inhibitors such as Novartis’ Cosentyx and Lilly’s Taltz, while theoretical concerns of suicidal ideation associated with suppressing more of the IL-17 pathway (sparked by Amgen’s IL-17RA inhibitor Siliq) have so far not played out. Therefore, strong efficacy with a fast onset of action and clean safety profile makes bimekizumab likely to be approved and an exciting new option for psoriasis patients. — Adam Schaffner, senior principal, InThought Research
Credit Suisse revenue forecasts: $308 million in psoriasis and $692 million in all indications by 2025
What physicians are saying: While Cosentyx was the first-in-class IL17 to market in psoriasis and has been very successful to date, recent Phase IIIB head-to head trials showed superiority to Cosentyx for PASI 100 at week 16 and week 48. It will be interesting to see how bimekizumab fares on the psoriasis market versus other IL17s and IL23s. — Nicola Bailey, SVP, head of autoimmune CoE, Ipsos
BMS-986165
Bristol Myers Squibb, Phase III
Indication: psoriasis
What clinical trials have found: BMS-986165 is an oral TYK2 inhibitor currently in Phase III trials for moderate to severe psoriasis. Phase II trials showed significant skin clearance after 12 weeks, while topline data from its first Phase III study announced in early November showed superiority to blockbuster drug Otezla. That’s a drug that BMS would have inherited through its acquisition of Celgene in 2019, but was instead sold to Amgen for $13 billion. That now seems like a worthwhile trade-off. The second Phase III data are expected in the Q1 2021.
InThought comment: BMS-986165 demonstrated impressive efficacy similar to IL-23 injectable drugs such as AbbVie’s Skyrizi and J&J’s Tremfya, but as an oral option it could be a game changer for patients with moderate-to-severe disease looking for a more convenient self-administration option. The drug has a lot of buzz around it as it is being pursued in other indications, including inflammatory bowel diseases. — Schaffner
Credit Suisse revenue forecast: $2.3 billion in all indications
What physicians are saying: BMS-986165 is touted as a future best-in-class oral drug with a new mechanism not seen in psoriasis. In addition to its oral convenience, it is exciting in that it is so far noted for a good side-effect profile, with possibly no sacrifice on efficacy. — Bailey
Etrasimod
Arena Pharmaceuticals, Phase III
Indication: ulcerative colitis
What clinical trials have found: Etrasimod is a once-daily oral S1P receptor modulator being examined for patients with moderate to severe ulcerative colitis, among other immune and inflammatory-mediated diseases. In the Phase II trial, patients taking a 2mg dose of etrasimod daily showed significant improvements in clinical remission over 12 weeks, while safety results supported a potential best-in-class profile.
InThought Comment: Phase II data was promising for BMS’s ozanimod, an oral drug with the same mechanism tackling a difficult IBD indication with no safety concerns. Greater curiosity exists for etrasimod compared to competitors in its class because it is being developed in more indications, including atopic dermatitis and alopecia areata. — Schaffner
Credit Suisse revenue forecast: $2.2 billion across IBD indications
What physicians are saying: Etrasimod will be the first non-JAK inhibitor oral available for UC. — Bailey
Blood Disorders
Most anticipated products
LentiGlobin
Bluebird Bio, pre-registration
Indication: sickle cell disease and beta thalassemia
What the clinical trials have found: LentiGlobin is the first gene therapy designed to increase levels of hemoglobin. The company is pursuing U.S. and European approval in sickle cell disease (SCD) in 2021, and also a U.S. approval in beta thalassemia.
For SCD, Group C of the HGB-206 study demonstrated a 99% reduction in annualized rate of vaso-occlusive crisis (VOC) and acute chest syndrome (ACS) in patients who had at least six months of follow-up and a history of VOCs or ACS. In beta thalassemia, the Northstar-2 (HGB-207) study for non-β0/β0 genotyped patients showed that 89% achieved primary endpoint of transfusion independence.
GlobalData consensus forecast: $1.25 billion in 2026
InThought comment: A potential one-time treatment leading to a cure would mean a significant improvement over all current available options in both sickle cell disease and beta thalassemia. The lentiviral vector that delivers the gene seems to be working as expected, with expression of the replacement β-globin gene HbAT87Q present at therapeutic levels (44% to 59%) in all patients. — Michelle Rivera, senior analyst, InThought Research
What physicians are saying: LentiGlobin offers physicians a unique treatment option that is expected to increase hemoglobin levels and reduce the number of pain crises patients experience, subsequently impacting their overall quality of life. — Nicole Allie, research manager, healthcare, Ipsos
EtranaDez (AMT-061)
UniQure/CSL Behring, Phase III
Indication: hemophilia B
What the clinical trials have found: UniQure/CSL Behring’s EtranaDez (etranacogene dezaparvovec/AMT-061) could be the first gene therapy approved for patients with hemophilia B, which is a rare blood clotting deficiency. EtranaDez consists of an AAV5 viral vector carrying a gene cassette with the Padua gene variant of Factor IX — an important blood clotting protein. Phase III topline data is expected by end of 2020 and a BLA filing is expected in 2021. Phase IIb data showed that all patients had stabilized and sustained Factor IX activity at functional curative levels after a single administration.
InThought comment: Its key advantage is that it is a one-time therapy that will replace current factor replacement or monoclonal antibody therapy, which requires infusions/injection regimes ranging from every other day to once a month. It will also provide stable FVIII or FIX levels, which will provide better outcomes in terms of bleed rates. — Chris Martin, senior principal, InThought Research
GlobalData consensus forecast: $695 million in 2026
What physicians are saying: Despite the high price tag of gene therapies, it is expected that the single administration will be as cost-effective as current weekly treatments. It offers an exciting new horizon in hemophilia therapy. — Isabel Palau, associate director, Ipsos
Roctavian (valoctocogene roxaparvovec)
Biomarin, Phase III, pre-registration
Indication: hemophilia A
What the clinical trials have found: Roctavian is aiming to be the first gene therapy approved for hemophilia A. Four-year data released in May 2020 showed cumulative mean annualized bleed rates (ABR) remain less than one in two cohorts and below pre-treatment baseline levels. The FDA granted breakthrough therapy designation for Roctavian and, while a launch was expected in 2021, the FDA has now requested another year of data. It could be available to patients before the end of 2022.
InThought comment: Roctavian’s complete response letter requested two-year data, versus the six-month data they had initially. So it is unlikely that Roctavian will receive approval in the U.S. in 2021, but it could still be approved in Europe. Similarly to EtranaDez, many discuss gene therapy as a long-term “holiday” from having hemophilia. However, there are four main issues for both these therapies, including durability, interpatient variability, high cost and the potential development of AAV neutralizing antibodies, which may affect eligibility to receive other gene therapies if durability is limited. — Martin
GlobalData consensus forecast: $1.05 billion in 2026
What physicians are saying: If successful, Roctavian could be a life-changing treatment for patients with hemophilia A, as most patients with severe hemophilia currently receive prophylaxis treatment with injections. — Palau
Other
Most anticipated products
Cardiovascular
Vericiguat
Merck, pre-registration
Indication: HFrEF (heart failure with reduced ejection fraction)
What the clinical trials found: While earlier Phase II trials in HFrEF failed, Bayer and Merck enrolled more than 5,000 high-risk patients into its Phase III outcomes trial (Victoria). It showed that vericiguat reduced cardiovascular death and hospitalization due to heart failure with a reduced relative risk of heart failure by 11 months. The drug’s PDUFA approval date is January 20.
InThought comment: Despite prior stumbles, vericiguat’s absolute risk reduction with a lack of excess adversity, such as hypotension, should make it approvable in a narrow yet very well defined high-risk population. — Henderson-MacLennan
Credit Suisse forecast: $1.8 billion by 2025
What physicians are saying: Eyes are on vericiguat after the Merck/Bayer co-filing gained FDA priority review and it demonstrated safety and efficacy in the Victoria trial. With anticipated market entry in early 2021, it will enter a crowded market where it will need to fight for market share versus more established products. However, if successful at defining its target patient population — one that is not currently well served by current treatment — it may prove a welcome addition to the treatment armory. — Jo Appleton, global head of client programs, healthcare, Ipsos
Women’s Health
Relugolix
Myovant, pre-registration
Indication: uterine fibroids
What the clinical trials found: In 2019, Myovant produced two successful clinical trials of relugolix combination therapy for heavy bleeding associated with uterine fibroids. While efficacy in reducing menstrual bleeding was strong, what was more impressive was the lack of bone mineral density loss, which is common with this class of therapies. The company is expecting an approval verdict on June 1.
InThought comment: Myovant’s entry poses a competitive threat to AbbVie’s direct competitor Oriahnn. While both have similar efficacy, Relugolix may have an edge with its once-daily dosing and lesser effects on reducing bone mineral density. Whether or not this is enough to drive switching remains to be seen. — Rivera
Credit Suisse revenue forecast: $500 million for uterine fibroids and $1.15 billion for all indications, including prostate cancer, by 2025
Rare Disease
Pegunigalsidase alfa (PRX-102)
Protalix, pre-registration
Indication: Fabry disease
What the clinical trials found: PRX-102 is an enzyme replacement therapy that replenishes the alpha-galactosidase A enzyme that is defective in Fabry disease, which is a life-threatening genetic disorder. Interim data from its Phase III Bridge study showed a substantial improvement in renal function in patients who switched from the currently used ERT Replagal, marketed by Takeda. The PDUFA date is January 27.
InThought comment: As a longer-acting version of an old-school enzyme replacement therapy (ERT), PRX-102 will be an easy plug-in with a captive audience of current ERT users. An approval decision should be straightforward and the drug should garner a healthy split of new starters and Replagal switchovers. — Henderson-MacLennan
What physicians are saying: Currently many patients don’t receive ERT, so PRX-102 should provide physicians with a valuable new option that importantly offers superior renal function improvements over current ERTs. — Helen Cox, senior client director, Ipsos
From the December 01, 2020 Issue of MM+M - Medical Marketing and Media
