According to recent EvaluatePharma analysis, Dupixent is forecasted to generate sales
of $4.56 billion in 2022 based on its anticipated 2017 launch. “ … a key worry now is
whether the manufacturing problems that
recently delayed Sanofi;Regeneron’s rheumatoid arthritis project sarilumab might also
hit Dupixent,” the analysts noted.
Manufacturing deficiencies were raised
by the FDA during a routine Current Good
Manufacturing Practice (CGMP) inspection
of a Sanofi manufacturing facility, which conducts “fill and finish” activities. Sanofi provided comprehensive responses to the U.S.
regulatory agency for the cited deficiencies.
Another Sanofi product expected to reach
blockbuster sales is Soliqua (insulin glargine
100 units;mL ; lixisenatide 33 mcg;mL
injection). The new product gained FDA
approval on Nov. 21, 2016, and was made
available by prescription in U.S. pharmacies
during early January. Soliqua 100; 33 is indicated for treating adults with type 2 diabetes
inadequately controlled on basal insulin (less
than 60 units daily) or lixisenatide. Soliqua
100; 33 joins together the company’s blockbuster diabetes brand Lantus and the GLP-1
receptor agonist lixisenatide. The once-a-day
injection was studied in a Phase III program
consisting of more than 1,900 patients.
In an insulin intensification study,
once-daily Soliqua 100; 33 showed statistical superiority for the change in HbA1c
from baseline to week 30 (p;0.0001) versus
Lantus, the most prescribed basal insulin
worldwide. Soliqua 100; 33 is delivered via
a single pre-filled SoloStar pen with a dose
range spanning 15 to 60 units and two starting doses to support patients’ insulin needs.
According to Sanofi, SoloStar technology is
the most frequently used disposable insulin
injection pen platform worldwide.
The European Commission granted marketing authorization in Europe for Suliqua
during January 2017 as a once-daily titratable fixed-ratio combination of basal insulin
glargine 100 units;mL and GLP-1 receptor
agonist lixisenatide for treating adults with
type 2 diabetes. The marketing authorization
is applicable to the 28 EU member states,
Iceland, Liechtenstein and Norway. Launches in individual EU countries were expected
from second-quarter 2017 onward.
Another expected future blockbuster mak-
ing its way into the marketplace during 2017
is the interleukin- 6 (IL- 6) receptor antibody
Kevzara;Kevsara. On Feb. 1, 2017, Sanofi and
Regeneron announced that Health Canada
approved Kevsara (sarilumab) for the treat-
ment of adult patients with moderately to
severely active rheumatoid arthritis who have
had an inadequate response or intolerance
to one or more biologic or non-biologic dis-
ease-modifying anti-rheumatic drugs.
A fully human monoclonal antibody,
Kevsara binds specifically to soluble and
membrane-bound IL- 6 receptors, and has
been demonstrated to inhibit IL-6-mediat-
ed signaling through these receptors. Local
production of IL- 6 by synovial and endo-
thelial cells in joints affected in chronic in-
flammatory disease, including rheumatoid
arthritis, may play a significant role in de-
velopment of the inflammatory processes.
The refiling of the sarilumab BLA to the
FDA is expected during first-quarter 2017.
This resubmission is subject to successful
completion of an FDA inspection of Sanofi’s Le Trait fill and finish facility, with an
expected action date in the second quarter.
The European Medicines Agency accepted for review the marketing authorization
application for sarilumab during July 2016
and a decision is expected in 2017.
During September 2016, the marketing
authorization application of SAR342434
(insulin lispro) was accepted for regulatory
review in the European Union for the treatment of diabetes.
The drive to become a leading biotech- nology company concentrated on in- novative approaches to treat rare and
specialty diseases shapes the foundation of
Shire’s R;D platform. The company’s expertise in discovering new therapies for rare,
life-threatening genetic diseases as well as
in developing medicines for patients treated by specialist physicians demonstrates
Shire’s continued dedication to patients and
Shire’s pipeline received significant boosts
when the company completed its acquisition
of Baxalta in June 2016 at a price tag of about
; 32 billion as well as through the purchase of
Dyax, which was completed in January 2016
at a potential value of $6.5 billion.
Shire has two subcutaneous projects undergoing Phase III studies: lanadelumab,
which has been projected to become the
top-selling hereditary angioedema drug in
2022 at $1.07 billion, and a subcutaneous
version of the already-marketed product
Cinryze. “Data with lanadelumab are due
in the second quarter of 2017, which should
give a clue whether it can live up to the lofty
expectations,” according to analysis from
Also known as SHP643, the potential
best-in-class drug lanadelumab came to Shire
via the acquisition of Dyax. Lanadelumab is
a subcutaneous anti-plasma kallikrein monoclonal antibody intended for prophylactic use.
Shire is performing a single pivotal trial
of SHP643 (formerly D;-2930) together
with an open-label extension study for prophylaxis of hereditary angioedema with the
goal of FDA approval during 2018. Shire has
shown proof of concept for SHP643 in long-term prevention of hereditary angioedema
without any significant safety concerns so
far. The new product candidate has received
orphan drug designation and breakthrough
therapy designation from U.S. regulators.
A new drug application is under FDA
review for SHP465, a novel, long-acting,
triple-bead, mixed amphetamine salts formulation. The new drug candidate is being
assessed as a potential once-per-day treatment for attention-deficit;hyperactivity disorder. An FDA regulatory decision is expected by June 20, 2017, which is the designated
Prescription Drug User Fee Act (PDUFA)
Shire refiled the new drug application for
SHP465 in response to the FDA approvable
letter that requested further clinical trials
and classified the response as a Class 2 re-submission with a review goal of six months.
Shire carried out two Phase III trials and
one open-label Phase I study to support the
refiling, all of which successfully met their
primary and key secondary endpoints.
For SHP621, the company is conducting
a Phase III induction trial for eosinophilic esophagitis (EoE) and simultaneously
enrolling a Phase III treatment extension
study. Shire completed Phase II studies
that met co-primary endpoints as well as
an open-label extension trial. SHP621 was
granted breakthrough therapy designation
by FDA in May 2016 for treating EoE.
The novel biologic SHP647 is intended
as a treatment of inflammatory bowel dis-
ease and is the only anti-integrin directly
targeting MAdCAM-1 with a potentially
differentiated and improved safety profile
compared to current treatments. Shire ex-
pected to initiate Phase III development of
SHP647 during 2017.
Shire is planning two Phase III trials
with SHP620;maribavir for treating cytomegalovirus infection in transplant recipient patients. All clinical trials of maribavir
have demonstrated an acceptable safety
profile and potent antiviral activity including against strains of cytomegalovirus resistant or refractory to other anti-CMV agents.
Adynovate [Antihemophilic Factor
(Recombinant), PEGylated; received marketing approval from the Food and Drug
Administration for new indications during
December 2016. The new indications provide more hemophilia A patients with access to proven prophylaxis with a simple,
twice-weekly dosing schedule.
Adynovate gained marketing clearance
as a treatment for hemophilia A in pediatric patients younger than 12 years. U.S.
regulators additionally approved the product for use in surgical settings for adult and
pediatric patients. Adynovate is built on the
full-length Advate [Antihemophilic Factor
(Recombinant); molecule, a market-leading
hemophilia A treatment with more than 13
years of real-world patient experience.
Shire during December reported topline
results from a Phase III study of Vonvendi
;von Willebrand factor (Recombinant); to
treat bleeds in elective surgical settings for
adults with severe von Willebrand disease,
the most common inherited bleeding disorder. The product is an on-demand recombinant treatment for adults living with VWD
and replaces von Willebrand factor. The results will form the basis of a supplemental
new drug application to the FDA requesting
an expanded indication for Vonvendi.
Shire introduced Cuvitru [Immune Glob-
ulin Subcutaneous (Human), 20; Solution;
to the U.S. marketplace in November as the
first Subcutaneous 20; treatment option
without proline to treat adult and pediatric
patients (2 years of age and older) with pri-
FDA approved the product in September
as a subcutaneous immune globulin (IG)
replacement therapy for primary immuno-
deficiency, a group of more than 300 ge-
netic disorders in which part of the body’s
immune system is missing or functions
improperly, in some cases making it more
difficult to fend off infections.
Shire is expand operations in Cam-
bridge, Mass., establishing a rare disease
innovation hub that will further catalyze the
company’s serial innovation across a variety
of rare diseases as well as highly specialized
Note: Timings are approximated to the nearest quarter and where appropriate subject to regulatory approval
Key anticipated events in next 12 months
Proof of Concept
Hunter I T Phase 3
Top Line Data
Clinical trial results
Regulatory filing or