Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO™ (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease | Business Wire

NEW YORK &. CARMIEL, Israel–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX,

TASE:PLX) announced today that the U.S. Food and Drug Administration

(FDA) approved ELELYSO™ (taliglucerase alfa) for injection for paediatric

patients. ELELYSO is therefore now indicated for long-term enzyme

replacement therapy (ERT) for adult and paediatric patients with a

confirmed diagnosis of Type 1 Gaucher disease.

“The approval of ELELYSO to treat paediatric patients with Type 1 Gaucher

disease provides physicians another treatment option for this rare and

potentially debilitating disease,” said Rory O’Connor, Senior Vice

President, Global Medical Affairs, Global Innovative Pharma Business,

Pfizer Inc. “This paediatric indication, along with the recent

announcement that ELELYSO received kosher certification by the Orthodox

Union (OU), reinforces the ongoing commitment of Pfizer to addressing

the needs of the Gaucher community.”

The safety and efficacy of ELELYSO were assessed in fourteen paediatric

patients with Type 1 Gaucher disease in two clinical trials. The first

trial consisted of nine patients in a 12-month, multi-center,

double-blind, randomized study in treatment-naïve patients aged two to

13 years. At the end of the 12-month study, therapeutic efficacy of

ELELYSO was demonstrated, as measured by a decrease in spleen and liver

volume and an increase in platelet count. A second trial consisted of 5

paediatric patients aged 6 to 16 years who were switched from

imiglucerase to ELELYSO. The trial was a 9-month, multi-center,

open-label, single-arm study in patients who'd been receiving

treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60

units/kg every other week for a minimum of 2 years. ELELYSO was

administered for 9 months at the same dose as each patient’s previous

imiglucerase dose. If needed, adjustment of dosage was allowed during

the study in order to maintain stability of clinical parameters. Mean

spleen and liver volume, platelet count and haemoglobin value remained

stable through 9 months of ELELYSO treatment.

The recommended dosage of ELELSYO for treatment-naïve adult and

paediatric patients four years of age and older is 60 units per kg of

body weight administered every other week as a 60 to 120 minute

intravenous infusion.

Patients previously treated on a stable dosage of imiglucerase are

recommended to begin treatment with ELELYSO at that same dosage when

they switch from imiglucerase to ELELYSO. Dosage adjustments can be made

based on achievement and maintenance of each patient’s therapeutic goals.

Serious hypersensitivity reactions, including anaphylaxis, have occurred

in some patients treated with ELELYSO. The most common adverse reactions

for ELELYSO in clinical trials were itching, flushing, headache, joint

pain, pain in extremity, abdominal pain, vomiting, fatigue, back pain,

dizziness, nausea and rash. Vomiting occurred more often in paediatric

patients than adults.

“While Type 1 Gaucher disease can manifest in childhood or adulthood,

the disease more often presents during childhood,” said Paige Kaplan,

MB, BCh, Section of Biochemical Genetics (Metabolic Diseases),

Children’s Hospital of Philadelphia. “it's important that children with

this disease have access to a range of FDA-approved treatment options

that are effective.”

As part of its ongoing commitment to helping address the unmet medical

needs of people with rare diseases, Pfizer also offers a specialized

support program —. Called Gaucher Personal Support (GPS) —. People

living with Gaucher disease. Pfizer GPS is a one-stop resource for

personalized patient support and speciality pharmacy services for

patients and healthcare professionals. Pfizer GPS employs a dedicated

and caring team of healthcare specialists who are available to help

patients with Gaucher disease and their caregivers with reimbursement

assistance, coordinating and locating infusion services and ongoing

pharmacy support. Using Pfizer GPS, patients and healthcare

professionals can access all support services 24/7 using one toll-free

phone number, 1-855-ELELYSO (1-855-353-5976).

ELELYSO for injection is supplied as 200 units per vial and is available

by prescription only.

For more information about Gaucher disease, please visit www.ELELYSO.com.

INDICATION

ELELYSO™ is indicated for long-term enzyme replacement therapy (ERT) for

adult and pediatric patients with a confirmed diagnosis of Type 1

Gaucher disease.

IMPORTANT SAFETY INFORMATION

Serious hypersensitivity reactions including anaphylaxis have occurred

in some patients treated with ELELYSO (taliglucerase alfa) for

injection. Intravenous use. When treated with ELELYSO your doctor

should monitor you before and after infusion for reactions.

Medical support should be readily available when ELELYSO is given.

Discontinue ELELYSO immediately if you show signs or symptoms of

anaphylaxis during infusion and get immediate medical care. Signs and

symptoms of anaphylaxis included hives, low blood pressure, flushing,

wheezing, chest tightness, nausea, vomiting and dizziness.

Signs and symptoms of hypersensitivity included itching, swelling under

the skin, flushing, redness, rash, nausea, vomiting, cough, chest

tightness and throat irritation. These reactions occurred up to 3 hours

after the start of infusion.

Management of hypersensitivity reactions is based on the severity of the

reaction. Your doctor may manage the reactions by slowing or temporarily

stopping the infusion, and/or treating with medications such as an

antihistamine, a fever reducer and/or corticosteroids for mild

reactions. Treatment with antihistamines and/or corticosteroids prior to

infusion with ELELYSO may prevent these reactions from reoccurring. If

severe hypersensitivity reactions occur, immediately stop the infusion

of ELELYSO and get immediate medical care.

You should be carefully re-evaluated for treatment with ELELYSO if

serious or hypersensitivity reactions including anaphylaxis occur.

The most common adverse reactions for ELELYSO are itching, flushing,

headache, joint pain, pain in extremity, abdominal pain, vomiting,

fatigue, back pain, dizziness, nausea and rash. Vomiting occurred more

often in children than adults.

The recommended dosage of ELELYSO for adults and children who are 4

years of age and older and not taking another ERT is 60 units per kg of

body weight given every other week as a 60 to 120 minute intravenous

infusion.

As with all therapeutic proteins, including (enzyme replacement therapy)

ERTs, there is a possibility of developing antibodies to ELELYSO. The

relationship between developing antibodies and hypersensitivity

reactions isn't clear. Your doctor should monitor you for antibodies to

ELELYSO if you've developed antibodies or if you've experienced

hypersensitivity reactions to ELELYSO or other ERTs.

If you're pregnant. Plan to become pregnant, you should talk to your

doctor about potential benefits and risks.

you're encouraged to report negative side effects of prescription drugs

to the FDA. Visit www.fda.gov/medwatch,

or call 1-800-FDA-1088.

For full prescribing information click here.

The health information contained herein is provided for educational

purposes only and isn't intended to replace discussions with a health

care provider. All decisions regarding patient care must be made with a

health care provider, considering the unique characteristics of the

patient. This product information is intended only for residents of the

United States.

About Gaucher Disease

Gaucher disease is an inherited lysosomal storage disorder in humans

that affects an estimated 10,000 people worldwide and can cause severe

and debilitating symptoms, including: enlargement of the liver and

spleen, various forms of bone disease, easy bruising. Anaemia (a low

number of red blood cells).

Gaucher disease consists of varying degrees of severity. It's been

sub-divided into three subtypes –. Types 1, 2. 3 –. According to the

presence or absence of neurological involvement. Type 1, the most

common, is found at a higher frequency among individuals who are of

Ashkenazi Jewish ancestry.

Pfizer and Rare Diseases

Rare diseases are among the most serious of all illnesses and impact

millions of patients worldwide, representing an opportunity to apply our

knowledge and expertise to help make a significant impact in addressing

unmet medical needs. The Pfizer focus on rare diseases builds on more

than a decade of experience and a global portfolio of 22 medicines

approved worldwide that treat rare diseases in the areas of hematology,

neuroscience, inherited metabolic disorders, pulmonology. Oncology.

Pfizer Inc.: Working together for a healthier world®

At Pfizer, we apply science and our global resources to bring therapies

to people that extend and significantly improve their lives. We strive

to set the standard for quality, safety and value in the discovery,

development and manufacture of health care products. Our global

portfolio includes medicines and vaccines as well as many of the world’s

best-known consumer health care products. Every day, Pfizer colleagues

work across developed and emerging markets to advance wellness,

prevention, treatments and cures that challenge the most feared diseases

of our time. Consistent with our responsibility as one of the world’s

premier innovative biopharmaceutical companies, we collaborate with

health care providers, governments and local communities to support and

expand access to reliable, affordable health care around the world. For

more than 150 years, Pfizer has worked to make a difference for all who

rely on us. To learn more, please visit us at www.pfizer.com.

Protalix BioTherapeutics Inc.

Protalix is a biopharmaceutical company focused on the development and

commercialisation of recombinant therapeutic proteins expressed through

its proprietary plant cell-based expression system, ProCellEx®.

Protalix’s unique expression system presents a proprietary method for

developing recombinant proteins in a cost-effective, industrial-scale

manner. Protalix’s first product manufactured by ProCellEx,

taliglucerase alfa, was approved for marketing by the U.S. Food and Drug

Administration (FDA) in May 2012, by Israel’s Ministry of Health in

September 2012, by the Brazilian National Health Surveillance Agency

(ANVISA) in March 2013, by the Mexican Federal Commision for the

Protection against Sanitary Risk (COFEPRIS) in April 2013, by the

Australian Therapeutic Goods Administration (TGA) in May 2014 and by the

regulatory authorities of other countries. Marketing applications for

taliglucerase alfa have been filed in additional territories as well.

Protalix has partnered with Pfizer Inc. for the worldwide development

and commercialisation of taliglucerase alfa, excluding Israel and

Brazil, where Protalix retains full rights. Protalix’s development

pipeline includes the following product candidates: PRX-102, a modified

version of the recombinant human alpha-GAL-A protein for the treatment

of Fabry disease. PRX-112, an orally-delivered glucocerebrosidase enzyme

that's produced and encapsulated within carrot cells. The treatment

of Gaucher disease. Pr-antiTNF, a similar plant cell version of

etanercept (Enbrel®) for the treatment of certain immune and

inflammatory diseases, such as rheumatoid arthritis, Crohn’s disease,

colitis, psoriasis and other autoimmune and inflammatory disorders.

PRX-110 for the treatment of Cystic Fibrosis. Others.

Protalix Forwards Looking Statement Disclaimer

To the extent that statements in this press release aren't strictly

historical, all such statements are forward-looking. Are made

pursuant to the safe-harbor provisions of the Private Securities

Litigation Reform Act of 1995. The terms “anticipate,”. “believe,”.

“estimate,”. “expect,”. “plan”. “intend”. Other words or phrases of

similar import are intended to identify forward-looking statements. Drug

discovery and development involve a high degree of risk. Factors that

might cause material differences include, among others: failure or delay

in the commencement or completion of our preclinical studies and

clinical trials which may be caused by several factors, including: risks

related to the commercialisation efforts for taliglucerase alfa in the

United States, Israel, Brazil and other countries in which it's

approved for sale. Risks relating to the review process of other foreign

regulatory and other governmental bodies. Risks relating to delays in

other foreign regulatory authorities’. Approval of any applications filed

for taliglucerase alfa or refusals to approve such filings, as well as

the decisions of such regulatory authorities regarding labelling and

other matters that could affect the availability of taliglucerase alfa

or its commercial potential. The risk that applicable regulatory

authorities may refuse to approve the marketing and sale of a drug

product even after acceptance of an application filed for the drug

product. The dependence on performance by third party providers of

services and supplies relating to the commercialisation of taliglucerase

alfa. The inherent risks and uncertainties in developing drug platforms

and products of the type we're developing. The impact of development of

competing therapies and/or technologies by other companies and

institutions. Potential product liability risks. Risks of securing

adequate levels of product liability and other necessary insurance

coverage. Other factors described in our filings with the U.S.

Securities and Exchange Commision. These forward-looking statements are

based on current information that may change and you're cautioned not

to place undue reliance on these forward-looking statements. The

statements in this release are valid only as of the date hereof and we

disclaim any obligation to update this information. All

forward-looking statements are qualified in their entirety by this

cautionary statement.

Pfizer Disclosure Notice

The information contained in this release is as of August 28, 2014.

Pfizer assumes no obligation to update forward-looking statements

contained in this release as the result of new information or future

events or developments.

This release contains forward-looking information about ELELYSO ™

(taliglucerase alfa) and about an indication in the U.S. for ELELYSO for

the long-term treatment of paediatric patients with a confirmed diagnosis

of Type 1 Gaucher disease, including their potential benefits, that

involves substantial risks and uncertainties that could cause actual

results to differ materially from those expressed or implied by such

statements. Risks and uncertainties include, among other things, the

uncertainties regarding the commercial success of ELELYSO and the

indication for paediatric use in the U.S.. Whether and when applications

will be filed for ELELYSO in other jurisdictions and whether and when

regulatory authorities in other jurisdictions will approve applications

for ELELYSO, as well as their decisions regarding labelling and other

matters that could affect the availability or commercial potential of

ELELYSO. Competitive developments.

A further description of risks and uncertainties can be found in

Pfizer’s Annual Report on Form 10-K for the fiscal year ended December

31, 2013 and in its subsequent reports on Form 10-Q, including in the

sections thereof captioned “Risk Factors”. “Forwards-Looking

Information That May Affect Future Results”, as well as in its

subsequent reports on Form 8-K, all of which are filed with the SEC and

available at www.sec.gov

and www.pfizer.com.

© 2014 Pfizer Inc. All rights reserved. TAL685104

Pfizer Inc.
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