In case you thought Covid-19 had slowed down US Food and Drug Administration’s New Drug Approvals, you’re in for a pleasant surprise — the FDA appears to be more active than ever before. By the end of June, the FDA had already approved 33 new drugs which put the approval activities within the ballpark of the past two years — 62 novel drugs were approved in 2018, while 54 were approved in 2019.
FDA’s Center for Drug Evaluation and Research (CDER) approved 25 new molecular entities and new therapeutic biological products, of which almost half — 12 out of 25 — were oncology drugs, while the rest of
the novel therapies were approved by the Center for Biologics Evaluation and Research.
The European Medical Agency (EMA) was also busy as the regulator issued a positive opinion for 41 drugs, of which 27 were classified as novel treatments.
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Conditional Approval for Gilead’s Remdesivir
Gilead’s Remdesivir has
certainly been one of the most talked about drugs
this year. While it is still under clinical evaluation, the FDA, EMA and the
Japanese Ministry of Health, Labour and Welfare (MHLW) found ways of providing
market access to this drug as a treatment against Covid-19.
On May 1, 2020, based on the totality of
scientific evidence available to the FDA, the
agency issued an Emergency Use Authorization (EUA), as it believed
that remdesivir may be effective
in treating Covid-19 and that the known and
potential benefits of remdesivir, when used to
treat Covid-19, outweigh the known and potential risks of such products.
On June 25, 2020, EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of conditional marketing authorization for Veklury (remdesivir).
The demand for remdesivir is such that the US bought more than 500,000 doses, which is all of Gilead’s production for July and 90 percent of production for August and September, leaving almost
no stock of remdesivir for the UK and Europe.
In 127 poor or middle-income countries, Gilead
is allowing generic drugmakers to supply remdesivir. It has signed non-exclusive voluntary licensing
agreements with generic pharmaceutical manufacturers based in Egypt, India and
Pakistan to further expand the supply of the antiviral drug.
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Vertex’s Kaftrio bags EMA approval
Earlier this year, PharmaCompass published its compilation of the sales forecasts for the new drugs approved by the FDA in 2019. The list was led by Vertex’s cystic fibrosis treatment — Trikafta — which is expected to have sales of US$ 3.935 billion by 2024.
Trikafta is a combination of ivacaftor, tezacaftor and elexacaftor and its stellar clinical data made the FDA approve the drug within three months of Vertex’s application filing and five months before FDA’s action date.
In June 2020, EMA’s CHMP adopted a positive opinion, recommending the granting of a marketing authorization for Vertex’s combination which will be marketed as Kaftrio.
EMA also adopted positive opinions on other
drugs which were previously approved by the FDA in
2019, such as Novartis’ Zolgensma and Piqray, Pfizer’s Staquis and Daurismo among many
others.
Immunomedic’s antibody-drug conjugate (ADC) — Trodelvy (sacituzumab govitecan-hziy) — was approved by the FDA for the treatment of adult patients with metastatic triple-negative breast cancer who have received at least two prior therapies for metastatic disease. Trodelvy follows remdesivir in our list of FDA approved drugs in 2020 with the highest sales potential. The current forecast for Trodelvy sales is US$ 2.151 billion by 2026.
FDA’s approval of Lundbeck’s Vyepti (eptinezumab) and Biohaven’s Nurtec ODT (rimegepant) for migraine
headaches brought additional CGRP-targeted products to the market. It will be
interesting to see how Nurtec ODT is accepted given it is a small molecule
drug, which makes administration easier. It was
recently promoted on social media by Khloe Kardashian.
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Covid-19 impacts drug launches
The pandemic has, however, started taking a
toll on drug launches. One of the most anticipated drug approvals of the year, Bristol-Myers Squibb’s multiple sclerosis (MS) treatment — Zeposia (ozanimod) — was approved in both the US and Europe. However, the launch of the drug would
be delayed due to the coronavirus outbreak.
The drug was added to BMS’s portfolio through its US$ 74 billion acquisition of Celgene last year. Its
approval was one of the three conditions set for a potentially higher payout
for Celgene investors.
Analysts have high hopes from ozanimod. Its
average peak sales for 2024 have been predicted to be at US$
1.62 billion by Cortellis, though the Covid-19 pandemic may weigh in there
as well.
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First non-statin cholesterol drug bags
FDA approval
This year also witnessed the first non-statin
treatment to be cleared for sale in the US in nearly 20 years. The drug, bempedoic acid, is made by Esperion Therapeutics Inc. This cholesterol-lowering drug is aimed at
helping millions of people who can’t tolerate or don’t get enough help from widely used statin pills like Lipitor and Crestor.
This new drug is to be used as an add-on treatment with statins. It lowers bad
cholesterol or low-density lipoprotein (LDL) by inhibiting its synthesis in the
liver. It targets patients with high cardiovascular risk.
Esperion also won approval of bempedoic acid
in combination with ezetimibe, another
cholesterol-lowering drug.
In January last year, Daiichi Sankyo Europe had entered into an exclusive licensing agreement with Esperion Therapeutics for Daiichi Sankyo Europe to market
bempedoic acid and bempedoic acid/ezetimibe combination tablet in the European
Economic Area and Switzerland.
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Approvals not granted to almost 20 drugs
There were setbacks too, and not everything
rolled smoothly. This year, almost 20 drug approvals were not granted. Among
the major setbacks were Bristol Myers Squibb and bluebird bio, Inc announcing
that they have received a Refusal to File letter from the FDA regarding the Biologics License
Application (BLA) for their CAR-T therapy, idecabtagene vicleucel (ide-cel), for patients with heavily pre-treated
relapsed and refractory multiple myeloma, which was
submitted in March 2020.
Upon preliminary
review, the FDA determined that the Chemistry, Manufacturing and Control (CMC)
module of the BLA requires further detail to complete the review. No additional
clinical or non-clinical data have been requested or are required.
Two years after Intarcia Therapeutics received
a CRL for its matchstick-sized, long-term drug
implant for type 2 diabetes, the FDA issued a second CRL to the company for its ITCA-650 implant. The
implant is designed to be a small, osmotic pump which can be slipped under the
skin and deliver a continuous, six-month dose of the
GLP-1 agonist exenatide.
The FDA also did not approve Intercept Pharmaceuticals’ obeticholic acid to treat NASH (or
nonalcoholic steatohepatitis, a liver condition in
which the buildup of fat
progressively scars the organ), as it wasn’t convinced that its benefits outweighed the potential risks.
After acquiring Allergan for US$ 63
billion, one of the first drugs which AbbVie was expecting
approval for was Abicipar pegol, their experimental DARPin therapy for patients with neovascular (wet) age-related macular degeneration (nAMD). However, FDA’s review indicated the rate of intraocular inflammation observed following administration of Abicipar pegol 2mg/0.05 mL
results in an unfavorable benefit-risk ratio in the treatment of wet AMD.
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Our view
While everyone’s attention is on the Covid-19 pandemic, the industry is certainly busy working towards getting new drugs to market. At the halfway mark, the FDA and EMA seem to be on track to set approval records this year, since the number of drugs approved by June are almost twice the number that were approved at the same time last year.
However, it remains to be seen how companies adapt their sales and marketing strategies in a world where mobility is likely to get restricted and interpersonal contact is set to reduce dramatically.
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Impressions: 69941
Acquisitions and spin-offs dominated headlines in 2019 and the tone was set very early with Bristol-Myers Squibb acquiring
New Jersey-based cancer drug company Celgene in a US$ 74 billion deal announced on
January 3, 2019. After factoring
in debt, the deal value ballooned to about US$ 95 billion, which according
to data compiled by Refinitiv, made it the largest healthcare deal on
record.
In the summer, AbbVie Inc,
which sells the world’s best-selling drug Humira, announced its acquisition of Allergan Plc, known for Botox and other cosmetic
treatments, for US$ 63 billion. While the companies are still awaiting
regulatory approval for their deal, with US$ 49 billion in combined 2019
revenues, the merged entity would rank amongst the biggest in the industry.
View Our Interactive Dashboard on Top drugs by sales in 2019 (Free Excel Available)
The big five by pharmaceutical sales — Pfizer,
Roche, J&J, Novartis and Merck
Pfizer
continued
to lead companies by pharmaceutical sales by reporting annual 2019 revenues of
US$ 51.8 billion, a decrease of US$ 1.9 billion, or 4 percent, compared to
2018. The decline was primarily attributed to the loss of exclusivity of Lyrica in 2019,
which witnessed its sales drop from US$ 5 billion in 2018 to US$ 3.3 billion in
2019.
In 2018, Pfizer’s then incoming CEO Albert Bourla had mentioned that the company did not see the need for any large-scale M&A activity as Pfizer had “the best pipeline” in its history, which needed the company to focus on deploying its capital to keep its pipeline flowing and execute on its drug launches.
Bourla stayed true to his word and barring the acquisition of Array Biopharma for US$ 11.4 billion and a spin-off to merge Upjohn, Pfizer’s off-patent branded and generic established medicines business with
Mylan, there weren’t any other big ticket deals which were announced.
The
Upjohn-Mylan merged entity will be called Viatris and is expected to have 2020
revenues between US$ 19 and US$ 20 billion
and could outpace Teva to
become the largest generic company in the world, in term of revenues.
Novartis, which had
followed Pfizer with the second largest revenues in the pharmaceutical industry
in 2018, reported its first full year earnings after spinning off its Alcon eye
care devices business division that
had US$ 7.15 billion in 2018 sales.
In 2019,
Novartis slipped two spots in the ranking after reporting total sales of US$
47.4 billion and its CEO Vas Narasimhan continued his deal-making spree by buying New
Jersey-headquartered The Medicines Company (MedCo) for US$ 9.7
billion to acquire a late-stage cholesterol-lowering
therapy named inclisiran.
As Takeda Pharmaceutical Co was
busy in 2019 on working to reduce its debt burden incurred due to its US$ 62
billion purchase of Shire Plc, which was announced in 2018, Novartis also purchased
the eye-disease medicine, Xiidra, from the Japanese drugmaker for US$ 5.3 billion.
Novartis’ management also spent a considerable part of 2019 dealing with data-integrity concerns which emerged from its 2018 buyout of AveXis, the
gene-therapy maker Novartis had acquired for US$ 8.7 billion.
The deal gave Novartis rights to Zolgensma,
a novel treatment intended for children less than two years of age with the
most severe form of spinal muscular atrophy (SMA). Priced at US$ 2.1 million,
Zolgensma is currently the world’s most expensive drug.
However,
in a shocking announcement, a month after approving the drug, the US Food and
Drug Administration (FDA) issued a press release on
data accuracy issues as the agency was informed by AveXis that
its personnel had manipulated data which
the FDA used to evaluate product comparability and nonclinical (animal)
pharmacology as part of the biologics license application (BLA), which was
submitted and reviewed by the FDA.
With US$
50.0 billion (CHF 48.5 billion) in annual pharmaceutical sales, Swiss drugmaker
Roche came in at number two position in 2019
as its sales grew 11 percent driven by
its multiple sclerosis medicine Ocrevus, haemophilia drug Hemlibra and cancer medicines Tecentriq and Perjeta.
Roche’s newly introduced medicines generated US$ 5.53 billion (CHF 5.4 billion) in growth, helping offset the impact of the competition from biosimilars for its three best-selling drugs MabThera/Rituxan, Herceptin and Avastin.
In late 2019, after months of increased
antitrust scrutiny, Roche completed
its US$ 5.1 billion acquisition of Spark Therapeutics to strengthen its presence in
gene therapy.
Last year, J&J reported almost flat worldwide sales of US$ 82.1 billion. J&J’s pharmaceutical division generated US$ 42.20 billion and its medical devices and consumer health divisions brought in US$ 25.96 billion and US$ 13.89 billion respectively.
Since J&J’s consumer health division sells analgesics, digestive health along with beauty and oral care products, the US$ 5.43 billion in consumer health sales from over-the-counter drugs and women’s health products was only used in our assessment of J&J’s total pharmaceutical revenues. With combined pharmaceutical sales of US$ 47.63 billion, J&J made it to number three on our list.
While the sales of products like Stelara, Darzalex, Imbruvica, Invega Sustenna drove J&J’s pharmaceutical business to grow by 4 percent over 2018, the firm had to contend with generic competition against key revenue contributors Remicade and Zytiga.
US-headquartered Merck, which is known as
MSD (short for Merck Sharp & Dohme) outside the United States and
Canada, is set to significantly move up the rankings next year fueled by its
cancer drug Keytruda, which witnessed a 55
percent increase in sales to US$ 11.1 billion.
Merck reported total revenues of US$ 41.75 billion and also
announced it will spin off its women’s health drugs,
biosimilar drugs and older products to create a new pharmaceutical
company with US$ 6.5 billion in annual revenues.
The firm had anticipated 2020 sales between US$ 48.8 billion and US$ 50.3 billion however this week it announced that the coronavirus pandemic will reduce 2020 sales by more than $2 billion.
View Our Interactive Dashboard on Top drugs by sales in 2019 (Free Excel Available)
Humira holds on to remain world’s best-selling drug
AbbVie’s acquisition of Allergan comes as the firm faces the expiration of patent protection for Humira, which brought in a staggering US$ 19.2 billion in sales last year for
the company. AbbVie has failed to successfully acquire or develop a major new
product to replace the sales generated by its flagship drug.
In 2019, Humira’s US revenues increased 8.6 percent to US$ 14.86 billion while internationally, due
to biosimilar competition, the sales dropped 31.1 percent to US$ 4.30 billion.
Bristol Myers Squibb’s Eliquis, which is also marketed by Pfizer, maintained its number two position
and posted total sales of US$ 12.1 billion, a 23 percent increase over 2018.
While Bristol Myers Squibb’s immunotherapy treatment Opdivo, sold in partnership with Ono in Japan, saw sales increase from US$ 7.57 billion to US$ 8.0 billion, the growth paled in comparison to the US$ 3.9
billion revenue increase of Opdivo’s key immunotherapy competitor Merck’s Keytruda.
Keytruda took the number three spot in drug sales that
previously belonged to Celgene’s Revlimid, which witnessed a sales decline from US$ 9.69 billion to US$ 9.4 billion.
Cancer treatment Imbruvica, which is marketed
by J&J and AbbVie, witnessed a 30 percent increase in sales. With US$ 8.1
billion in 2019 revenues, it took the number five position.
View Our Interactive Dashboard on Top drugs by sales in 2019 (Free Excel Available)
Vaccines – Covid-19 turns competitors into partners
This year has been dominated by the single biggest health emergency in years — the novel coronavirus (Covid-19) pandemic. As drugs continue to fail to meet expectations, vaccine development has received a lot of attention.
GSK reported the highest vaccine sales of all drugmakers with
total sales of US$ 8.4 billion (GBP 7.16 billion), a significant portion of its
total sales of US$ 41.8 billion (GBP 33.754 billion).
US-based Merck’s vaccine division also reported a significant increase in sales to US$ 8.0 billion and in 2019 received FDA and EU approval to market its Ebola vaccine Ervebo.
This is the first FDA-authorized vaccine against the deadly virus which causes
hemorrhagic fever and spreads from person to person through direct contact with
body fluids.
Pfizer and Sanofi also reported an increase in their vaccine sales to US$ 6.4
billion and US$ 6.2 billion respectively and the Covid-19 pandemic has recently
pushed drugmakers to move faster than ever before and has also converted
competitors into partners.
In a rare move, drug behemoths — Sanofi and GlaxoSmithKline (GSK) —joined hands to develop a vaccine for the novel coronavirus.
The two companies plan to start human trials
in the second half of this year, and if things go right, they will file
for potential approvals by the second half of 2021.
View Our Interactive Dashboard on Top drugs by sales in 2019 (Free Excel Available)
Our view
Covid-19 has brought the world economy to a grinding halt and shifted the global attention to the pharmaceutical industry’s capability to deliver solutions to address this pandemic.
Our compilation shows that vaccines and drugs
for infectious diseases currently form a tiny fraction of the total sales of
pharmaceutical companies and few drugs against infectious diseases rank high on
the sales list.
This could well explain the limited range of
options currently available to fight Covid-19. With the pandemic currently infecting
over 3 million people spread across more than 200 countries, we can safely
conclude that the scenario in 2020 will change substantially. And so should our
compilation of top drugs for the year.
View Our Interactive Dashboard on Top drugs by sales in 2019 (Free Excel Available)
Impressions: 55099
The US Food and Drug Administration’s (FDA) momentum of new drug approvals dropped marginally in 2019 — while 62 novel drugs were approved in 2018, 54 were approved in 2019. FDA’s Center for Drug Evaluation and Research (CDER) approved 48 drugs while another six were approved by the Center for Biologics Evaluation and
Research (CBER).
Novartis dominated new drug approvals as CDER approved five of its new drugs across five different therapeutic areas. But then, it also made headlines when the FDA raised data accuracy issues over the Swiss drugmaker’s gene therapy — Zolgensma — which sent the company into a crisis
management mode.
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The gene therapy drug
came into the fold of Novartis in April 2018 through the US$ 8.7 billion acquisition of Illinois-based AveXis Inc.
Zolgensma — the world’s most expensive drug — was approved as a one-time treatment for spinal muscular atrophy (SMA) in May 2019.
However, in March 2019, Novartis learnt of manipulation in the raw data derived
from a mice assay used at the time to test Zolgensma samples, but only alerted the FDA in June — about a month after the drug gained US approval.
Post that, Novartis came under fire from US lawmakers who said the company should have informed the regulators about the data irregularities before the drug’s approval in May, instead of waiting to conclude an internal investigation.
Novartis’ other approvals were brolucizumab (for treatment of macular
degeneration), siponimod (to treat multiple sclerosis), alpelisib (breast cancer), crizanlizumab (sickle
cell disease) and triclabendazole (fascioliasis, a tropical disease).
View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)
Vertex’s Trikafta leads in estimated sales potential
At PharmaCompass,
we compiled the sales forecasts for the new drugs approved by the FDA in 2019. Vertex’s cystic fibrosis treatment — Trikafta — with expected sales of US$ 3.935 billion by 2024, leads our list.
Trikafta is a combination of ivacaftor, tezacaftor and elexacaftor. Vertex already has three cystic fibrosis drugs on the market — Kalydeco (a standalone ivacaftor treatment), Symdeko (a combination of ivacaftor and tezacaftor) and Orkambi (which contains ivacaftor and lumacaftor).
Trikafta’s stellar clinical data made the FDA approve the drug within three months of Vertex’s application filing and five months before FDA’s action date.
Abbvie’s Humira, the top-selling drug in the world, is beginning to face generic competition in certain parts of the world and two new drug approvals in 2019 — Skyrizi (risankizumab for
plaque psoriasis) and Rinvoq (upadacitinib for rheumatoid arthritis) — are critical to AbbVie's plans to
grow beyond Humira. The two drugs are expected to contribute over US$ 5.5 billion by 2024 to AbbVie’s top-line.
In April, AstraZeneca struck a deal worth US$ 6.9 billion with Japanese drug major
Daiichi Sankyo, to jointly develop and commercialize the antibody drug conjugate cancer therapy —trastuzumab deruxtecan. The
breast cancer treatment won FDA approval in December and analysts forecast
sales of US$ 2.5 billion by 2024 with some believing that the drug could
achieve US$ 7 billion in peak sales.
View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)
The rise of new drugs from Asia
While Daiichi won the FDA nod for its cancer treatment — Turalio (pexidartinib) — new drugs from established Japanese drug makers like Shionogi, Kyowa Kirin and Eisai also bagged the American agency’s approval.
Shionogi’s novel antibiotic — Fetroja (cefiderocol) — got approved for complicated urinary tract infections while Kyowa won the nod for its Parkinson’s treatment — Nourianz (istradefylline). Eisai’s insomnia drug — Dayvigo (lemborexant) — was one of the last drugs approved in the year and is expected to generate sales of US$ 329 million.
View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)
In 2019, the FDA also
approved new drugs from China and Korea. Zanubrutinib (branded
as Brukinsa) developed by Chinese biotech BeiGene won accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive and rare form of blood cancer.
This was followed, almost immediately, by news that the agency approved a new treatment for partial-onset seizures in adults. The drug — cenobamate — will be sold as Xcopri by SK Life Science,
the US subsidiary of the massive Korean conglomerate SK Group. Xcopri will
provide another therapeutic option to patients with epilepsy. This approval
marked the first time a Korean company independently took a compound from the
stage of discovery to FDA approval.
Xcopri is SK Life Science’s first commercial product and wraps a decade of quiet growth from SK Life Science, which has been developing its presence in the pharmaceutical industry.
The company plans to hire a salesforce to support the drug’s launch, which is expected to generate over US$ 1.5 billion in sales.
View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)
Sanofi’s controversial dengue vaccine, cast-off drug get approved
The FDA very narrowly approved Sanofi’s controversial dengue vaccine — Dengvaxia. The vaccine, which took 20 years to develop, got embroiled in a controversy when in 2017, Sanofi disclosed that Dengvaxia could increase the risk of severe dengue in children who had never been exposed to the virus.
In Philippines, 800,000 school-age children had been vaccinated against dengue, the world’s fastest growing infectious disease.
The year 2019
had begun with Bristol-Myers Squibb (BMS) and Celgene announcing their US$ 74 billion mega-merger. As the firms worked through the year to get the merger approved, the FDA signed off on Celgene’s myelofibrosis drug Inrebic
(fedratinib).
Inrebic — the once-daily oral drug — is the first new treatment in nearly a decade approved to treat myelofibrosis. The story of Inrebic’s rise from the dead is a major success for Celgene,
which picked up the drug as a part of a US$ 1.1 billion acquisition of Impact
Biomedicines in January 2018. Before that, Inrebic had passed through multiple
hands.
Fedratinib was a flop
for Sanofi as patients began to develop a dangerous neurological
condition tied to vitamin B deficiency called Wernicke’s encephalopathy. As a result, the FDA put a clinical hold on it in 2013 and Sanofi ultimately shelved the effort.
Executives
at BMS have said the drug is among a group of potential blockbusters at
Celgene.
View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)
Our view
With over 40 companies from across the world getting new
drugs approved in 2019 across 17 therapeutic areas, the horizon for new drug
development across the globe continues to widen.
Although 2018 was a landmark year for approvals, given all
the development activity underway, the day is not far when this record too will get beaten. And
2020 could well witness that day!
View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)
Impressions: 14610