- FDA sets target action date of October 19,
2019 -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
the U.S. Food and Drug Administration (FDA) has accepted for
priority review the company’s supplemental Biologics License
Application (sBLA) for ULTOMIRIS® (ravulizumab-cwvz), the company’s
long-acting C5 complement inhibitor, for the treatment of people
with atypical hemolytic uremic syndrome (aHUS) in order to inhibit
complement-mediated thrombotic microangiopathy (TMA). The FDA has
set a target action date of October 19, 2019 under the Prescription
Drug User Fee Act (PDUFA).
“This acceptance is an important step in our efforts to deliver
a potential new standard of care to people living with this
devastating disease,” said John Orloff, M.D., Executive Vice
President and Head of Research & Development at Alexion. “We
look forward to working closely with the FDA to facilitate a rapid
review of this application.”
Atypical HUS, also known as complement-mediated TMA, is a severe
and chronic ultra-rare disease that can cause progressive damage to
vital organs, predominantly the kidneys, leading to kidney failure
and premature death.
The sBLA is based on previously announced results, which were
recently presented at the European Renal Association – European
Dialysis and Transplant Association (ERA-EDTA) Congress, from the
Phase 3 study of ULTOMIRIS in people with aHUS, which met the
primary endpoint of complete TMA response, defined by hematologic
normalization and improved kidney function.
Important ULTOMIRIS Safety Information ULTOMIRIS®
(ravulizumab-cwvz) is a prescription medicine called a monoclonal
antibody. ULTOMIRIS is used to treat adults with a disease called
Paroxysmal Nocturnal Hemoglobinuria (PNH). It is not known if
ULTOMIRIS is safe and effective in children.
ULTOMIRIS is a medicine that affects the immune system.
ULTOMIRIS can lower the ability of the immune system to fight
infections. ULTOMIRIS increases the chance of getting serious and
life-threatening meningococcal infections. Meningococcal infections
may quickly become life-threatening and cause death if not
recognized and treated early.
Meningococcal vaccines must be received at least 2 weeks before
the first dose of ULTOMIRIS if one has not already had this
vaccine. If one’s doctor decided that urgent treatment with
ULTOMIRIS is needed, meningococcal vaccination should be
administered as soon as possible. If one has not been vaccinated
and ULTOMIRIS therapy must be initiated immediately, 2 weeks of
antibiotics should also be administered with the vaccinations. If
one had a meningococcal vaccine in the past, additional vaccination
might be needed before starting ULTOMIRIS. Call one’s doctor or get
emergency medical care right away if any of these signs and
symptoms of a meningococcal infection occur: headache with nausea
or vomiting, headache with a stiff neck or stiff back, fever and a
rash, muscle aches with flu-like symptoms, headache and fever,
fever, confusion, and eyes sensitive to light.
ULTOMIRIS is only available through a program called the
ULTOMIRIS REMS.
ULTOMIRIS may also increase the risk of other types of serious
infections. People who take ULTOMIRIS may have an increased risk of
getting infections caused by Streptococcus pneumoniae and
Haemophilus influenzae. Certain people may also have an increased
risk of gonorrhea infection. To find out if one is at risk for
gonorrhea infection, about gonorrhea prevention, and regular
testing, talk to the healthcare provider. Call the healthcare
provider right away if one has any new signs or symptoms of
infection.
Before one receives ULTOMIRIS, tell the doctor about all of the
medical conditions, including if one: has an infection or fever,
are pregnant or plan to become pregnant, and are breastfeeding or
plan to breastfeed. It is not known if ULTOMIRIS will harm an
unborn baby. It is not known if ULTOMIRIS passes into the breast
milk. One should not breast feed during treatment and for 8 months
after one’s final dose of ULTOMIRIS.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. ULTOMIRIS and other medicines can affect each other
causing side effects. Know the medications one takes and the
vaccines one receives. Keep a list of them to show the doctor and
pharmacist when one gets a new medicine.
If one stops receiving ULTOMIRIS, the doctor will need to
monitor closely for at least 16 weeks after one stops ULTOMIRIS.
Stopping ULTOMIRIS may cause breakdown of the red blood cells due
to PNH. Symptoms or problems that can happen due to red blood cell
breakdown include: drop in the number of the red blood cell count,
tiredness, blood in the urine, stomach-area (abdomen) pain, blood
clots, shortness of breath, trouble swallowing, and erectile
dysfunction (ED) in males.
ULTOMIRIS can cause serious side effects including infusion
reactions. Infusion reactions may happen during one’s ULTOMIRIS
infusion. Symptoms of an infusion reaction with ULTOMIRIS may
include lower back pain, pain with the infusion, or feeling faint.
Tell the doctor or nurse right away if these symptoms develop, or
any other symptoms during the ULTOMIRIS infusion that may mean one
is having a serious infusion reaction, including: chest pain,
trouble breathing or shortness of breath, swelling of the face,
tongue, or throat, and feel faint or pass out. One’s doctor will
treat the symptoms as needed. The most common side effects of
ULTOMIRIS are upper respiratory infection and headache.
Please see the full Prescribing Information and Medication Guide
for ULTOMIRIS, including Boxed WARNING regarding serious and
life-threatening meningococcal infections/sepsis.
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
diseases through the discovery, development and commercialization
of life-changing therapies. As the global leader in complement
biology and inhibition for more than 20 years, Alexion has
developed and commercializes two approved complement inhibitors to
treat patients with paroxysmal nocturnal hemoglobinuria (PNH) as
well as the first and only approved complement inhibitor to treat
atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine
receptor (AchR) antibody-positive generalized myasthenia gravis
(gMG), and is also developing it for patients with neuromyelitis
optica spectrum disorder (NMOSD). Alexion also has two highly
innovative enzyme replacement therapies for patients with
life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency
(LAL-D). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. Alexion focuses its research efforts on novel
molecules and targets in the complement cascade and its development
efforts on the core therapeutic areas of hematology, nephrology,
neurology, and metabolic disorders. Alexion has been named to the
Forbes’ list of the World’s Most Innovative Companies seven years
in a row and is headquartered in Boston, Massachusetts’ Innovation
District. The company also has offices around the globe and serves
patients in more than 50 countries. This press release and further
information about Alexion can be found at: www.alexion.com.
[ALXN-G]
Forward-Looking Statement This press release contains
forward-looking statements, including statements related to: the
belief that ULTOMIRIS has the potential to become the new standard
of care for patients with aHUS in order to inhibit
complement-mediated thrombotic microangiopathy; the Company will
work closely with FDA in order to facilitate a rapid review of this
sBLA; the Company’s plans to make future regulatory filings for
approval of certain products and product candidates and the timing
of such filings; potential future regulatory approval for the
marketing of ULTOMIRIS and other products and product candidates;
and the potential benefits of current products and products under
development and in clinical trials (including ULTOMIRIS as a
treatment for patients with PNH). Forward-looking statements are
subject to factors that may cause Alexion's results and plans to
differ materially from those forward-looking statements, including
for example: the FDA and other regulatory agencies do not approve
the use of ULTOMIRIS as a therapy for patients with aHUS in order
to inhibit complement-mediated thrombotic microangiopathy; the sBLA
and other applications for approval of products are not deemed
sufficient by the appropriate regulatory authorities and are not
approved (or require additional information or data which may be
time consuming to generate); delays (expected or unexpected) in the
time it takes regulatory agencies to review and make determinations
on applications for the marketing approval of our products;
inability to timely submit (or failure to submit) future
applications for regulatory approval for our products and product
candidates; any potential post-approval restrictions that the FDA,
European Commission, the EMA or any other regulatory agency may
impose on ULTOMIRIS; ULTOMIRIS and other products and product
candidates do not gain regulatory approval from the FDA, European
Commission, MHLW, EMA or other regulatory authorities; our
products, including ULTOMIRIS do not gain acceptance among patients
and/or physicians and do not become the standard of care for
certain indications; inability to timely initiate (or failure to
initiate) and complete future clinical trials due to safety issues,
IRB decisions, CMC-related issues, expense or unfavorable results
from earlier trials (among other reasons); our dependence on sales
from our principal product (SOLIRIS); our inability to facilitate
the timely conversion of PNH patients (and patient with any future
indications) from SOLIRIS to ULTOMIRIS; regulatory agencies do not
accept the proposed indications for our product label (or approves
a product with a for an indication that is for a limited patient
population); payer, physician and patient acceptance of ULTOMIRIS
as an alternative to SOLIRIS; appropriate pricing for ULTOMIRIS;
future competition from biosimilars and novel products (and that
this future competition causes, among other things, ULTOMIRIS not
to be the standard of care for certain indications); decisions of
regulatory authorities regarding the adequacy of our research,
marketing approval or material limitations on the marketing of our
products; delays or failure of product candidates to obtain
regulatory approval; delays or the inability to launch product
candidates due to regulatory restrictions, increased expense or
other matters; interruptions or failures in the manufacture and
supply of our products and our product candidates; failure to
satisfactorily address matters raised by the FDA, European
Commission, the EMA, MHLW and other regulatory agencies regarding
products and product candidates; results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger clinical trials (or in broader patient populations)
and do not ensure regulatory approval; the possibility that results
of clinical trials are not predictive of safety and efficacy and
potency of our products (or we fail to adequately operate or manage
our clinical trials) which could cause us to halt trials,
discontinue sales of our products, delay or prevent us from making
regulatory approval filings or result in denial of approval of our
product candidates; unexpected delays in clinical trials;
unexpected concerns regarding products and product candidates that
may arise from additional data or analysis obtained during clinical
trials (or following commercialization); future product
improvements may not be realized due to expense or feasibility or
other factors; the possibility that current rates of adoption of
our products are not sustained; the adequacy of our
pharmacovigilance and drug safety reporting processes; failure to
protect and enforce our data, intellectual property and proprietary
rights and the risks and uncertainties relating to intellectual
property claims, lawsuits and challenges against us (including
intellectual property lawsuits relating to ULTOMIRIS brought by
third parties and inter partes review petitions submitted by third
parties); the risk that third party payors (including governmental
agencies) will not reimburse or continue to reimburse for the use
of our products at acceptable rates or at all; the possibility that
expected tax benefits will not be realized; potential declines in
sovereign credit ratings or sovereign defaults in countries where
we sell our products; delay of collection or reduction in
reimbursement due to adverse economic conditions or changes in
government and private insurer regulations and approaches to
reimbursement; uncertainties surrounding legal proceedings, company
investigations and government investigations, including
investigations of Alexion by the U.S. Securities and Exchange
Commission (SEC) and U.S. Department of Justice; the risk that
estimates regarding the number of patients with PNH, aHUS, gMG, HPP
and LAL-D and other indications we are pursuing are inaccurate; the
risks of changing foreign exchange rates; and a variety of other
risks set forth from time to time in Alexion's filings with the
SEC, including but not limited to the risks discussed in Alexion's
Quarterly Report on Form 10-Q for the quarter ended March 31, 2019
and in our other filings with the SEC. Alexion disclaims any
obligation to update any of these forward-looking statements to
reflect events or circumstances after the date hereof, except when
a duty arises under law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190620005331/en/
Alexion: Media Megan Goulart, 857-338-8634 Senior
Director, Corporate Communications
Investors Susan Altschuller, Ph.D., 857-338-8788 Vice
President, Investor Relations
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