2017 was a landmark
year for the pharmaceutical industries in the US and Europe, with a sharp
increase in the number of new molecular entities (NMEs) being approved in both
the geographies.
The US Food and Drug Administration (USFDA) approved 46 NMEs
in 2017, a strong bounce back from only 22 NMEs approved in 2016.
The
46 approvals by the USFDA’s Center
for Drug Evaluation and Research (CDER) is the
second highest total since 1996 when 53 NMEs were approved.
In
Europe, the European Medicines Agency (EMA) approved 35 drugs with a new active
substance, up from 27 in 2016. We also identified eight drugs that got approved
in Europe before winning the USFDA nod.
This
week PharmaCompass shares its compilation of the 46 novel drugs approved
in the US, of which 34 (or 74 percent) were small molecules and 12 (or
26 percent) were biologics, which were approved by the USFDA.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
We
have also evaluated these new
drug therapy approvals with regard to their estimated sales potential. While
the sales estimates of a new drug are far from certain, our effort is directed
at providing insights into the potential these drugs hold along with attempting
to identify emerging market trends.
An
overview of USFDA’s approvals
The USFDA’s CDER focusses extensively on the approval of novel drugs “which are often among the more innovative products in the marketplace, and/or help advance clinical care by providing therapies” that have never before been marketed in the United States.
In addition, CDER
also looks at new and expanded uses of USFDA-approved drugs. In 2017, the CDER
also worked on approving five new biosimilar drugs, which are highly similar to
the USFDA- approved
therapeutic biological products.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
New formulations or
new manufacturers of USFDA-approved products that can provide advantages over
original products, such as being able to take the drug on an empty stomach and
not with food, were also approved.
New dosage forms
entered the market that add value to already approved drugs, such as chewable
tablets for patients unable to swallow pills and Abilify MyCite (aripiprazole
tablets with a sensor).
Aripiprazole was
first approved by the USFDA in 2002 as a tablet to treat patients with
schizophrenia and was marketed under the brand name Abilify.
In 2017, Abilify MyCite
was approved as a tablet which contains an electronic sensor. Abilify MyCite
allows the patient to track whether he or she has taken the medication via a
smartphone or the cloud.
Patients can also
give their caregivers or physicians access to the information through a
web-based portal.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
In the list of 2017 approvals, CDER identified 15 of the 46 novel drugs approved in 2017 (33 percent) as first-in-class while 18 drugs (39 percent) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans.
Of the 46 novel drugs
approved in 2017, 36 (78 percent) were approved in the United States before
receiving approval in any other country.
The American stars of 2017, by sales revenue
Of all the drugs
approved by the USFDA, the highest expectations are from Regeneron and Sanofi’s first-in-class monoclonal antibody (mAb) — dupilumab. The (IL)-4 receptor subunit-α antagonist has shown benefits in various kinds of inflammatory and allergic diseases.
While the USFDA approved the drug for atopic dermatitis, it is also being developed for asthma, chronic obstructive pulmonary disease (COPD) and other indications.
Evaluate Pharma analysts estimate the sales of dupilumab
at US$ 4,938 million by 2022.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
Expectations are also
high from two other mAb products. Genentech and Roche’s anti-CD20 mAb ocrelizumab for relapsing and primary
progressive forms of multiple sclerosis is expected to bring in US$ 4,088
million by 2022 while Pfizer’s avelumab, an anti-PD L1 cancer drug
co-developed along with Merck KGaA, which was approved to treat metastatic
Merkel cell carcinoma, is expected to achieve peaks sales of US$ 4-6 billion
with an estimated US$ 3 billion in annual sales for Pfizer.
In late 2015, AstraZeneca partnered with Acerta Pharma to develop a potential best-in-class irreversible oral Bruton’s tyrosine kinase (BTK) inhibitor — acalabrutinib.
AstraZeneca
acquired 55 percent equity in Acerta for an upfront payment of US$ 2.5 billion
and should have completed its payment of an additional US$ 1.5 billion now that
the USFDA has approved the cancer drug, which is estimated to have peak sales
of US$ 2,500 million.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
Also
expected to bring in US$ 2,500 million in sales is Novartis’ treatment for postmenopausal women with a type of advanced breast cancer — ribociclib. While the USFDA
approved the drug in March 2017, the EMA
approved it in August 2017.
The
European specials of 2017
While
many drugs were approved in the United States before being approved in Europe, PharmaCompass
has identified 8 active substances which have not been approved in the US but
got EMA approval in 2017.
AstraZeneca’s treatment for hyperkalaemia — Lokelma (sodium zirconium
cyclosilicate) — got approved by the EMA. However, the FDA did not approve it after the agency raised concerns over the manufacturing of the drug following an inspection of the facility.
Lutathera
— a nuclear medicine targeted at the type of cancer that killed former Apple Inc co-founder and CEO Steve Jobs — got approved by the EMA and its developer Advanced Accelerator Applications (AAA), who in turn got bought over by Novartis for US$ 3.9 billion.
The
application for Lutathera has been filed with the USFDA. Under the Prescription
Drug User Fee Act (PDUFA), the agency is scheduled to make a final approval
decision this week (on January 26, 2018).
Tivozanib
(Fotivda, an oral, once-daily, vascular endothelial growth factor or VEGF) tyrosine kinase inhibitor (TKI)
discovered by Kyowa Hakko Kirin and approved for
the treatment of adult patients with advanced renal cell carcinoma was also
approved in Europe before getting the nod in the US.
Our view
While the USFDA’s CDER approved the highest number of drugs in two decades, the approvals of the year were done by USFDA’s Center for Biologics Evaluation and Research (CBER) which approved two cell-based gene therapies.
Novartis’ Kymriah (tisagenlecleucel), the first-ever gene therapy — known as CAR-T (short for chimeric antigen receptor T-cell) — was approved for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
In its press statement, the USFDA described this as a “historic action”.
CAR-T is a type of cancer immunotherapy
that harnesses the body’s immune system to fight cancer cells. In this case, the therapy removes a person’s cells, reengineers them and then puts them back in their body to attack cancer cells.
In CAR-T therapy, every single dose of the
treatment is unique and completely personalized to the patient. The Novartis drug came with a list price of US$ 475,000 for a one-time treatment.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
Gilead also
bet big on the technology and acquired Kite Pharma for US$ 11.9 billion in
August. The USFDA also approved Kite’s most advanced CAR-T therapy candidate — axicabtagene ciloleucel (axe-cel).
Gilead announced a price significantly lower than the launch price at which Novartis
is selling its CAR-T treatment (Kymriah).
With Novartis and
AstraZeneca getting three therapies approved, Pfizer, Roche, Valeant and the
team of Sanofi and
Regeneron bagging two each
and almost half the drugs approved by the USFDA’s CDER in 2017 expected to generate more than US$ 1 billion in sales, things look promising for innovation in pharmaceuticals and also provide interesting
opportunities for generic drug manufacturers.
Click here for our list of FDA’s Novel Drug Approvals in 2017 (Excel version available) for FREE!
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