FDA Approves Updated Labeling for Gattex® (Teduglutide [rDNA origin]) for Injection to Include Long-Term Data

BEDMINSTER, N.J.--(BUSINESS WIRE)--

NPS Pharmaceuticals, Inc. NPSP, a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved updated labeling for Gattex® (teduglutide [rDNA origin]) for injection to include long-term data from the STEPS 2 study of adult patients with Short Bowel Syndrome (SBS). The data demonstrate that patients who continued Gattex treatment beyond two years achieved clinically meaningful reductions in parenteral support requirements, including volume and days off, with 13 out of 88 patients across all groups achieving complete independence.

In the U.S., Gattex is indicated for the treatment of adult patients with SBS who are dependent on parenteral support. Full prescribing information for Gattex is available by clicking here or visiting www.Gattex.com.

“We are pleased that the FDA has approved updated labeling for Gattex, as it provides important information for healthcare professionals and patients about long-term use of Gattex therapy,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharma. “The STEPS 2 study demonstrated that there was an increased response to treatment over time in all groups receiving Gattex in terms of volume reductions, days off and complete weaning of parenteral support.”

About STEPS 2

The efficacy, safety, and tolerability of Gattex was evaluated in a randomized, double-blind, placebo-controlled, parallel-group, multi-national, multi-center clinical trial, known as STEPS, in adults with SBS who were dependent on parenteral nutrition/intravenous (PN/I.V.) support for at least 12 months and required PN at least 3 times per week. STEPS 2 was a 2-year open-label extension of STEPS in which 88 subjects received Gattex 0.05 mg/kg/day. Ninety-seven percent (76/78) of subjects who completed STEPS elected to enroll in STEPS 2 (37 received Gattex; 39 received Placebo). An additional 12 subjects entered STEPS 2, who had been optimized and stabilized but not randomized in STEPS because of closed enrollment.

30 months exposure

Thirty Gattex subjects completed a total duration of 30 months (STEPS followed by STEPS 2 treatment). Of these, 28 subjects (93%) achieved a 20% or greater reduction of parenteral support. Of responders in STEPS who had completed 2 additional years of continuous treatment with Gattex, 96% (21/22) sustained their response to Gattex. The mean reduction in PN/I.V. (n=30) was 7.55 L/week (a 65.6% reduction from baseline). Ten subjects were weaned off their PN/I.V. support while on Gattex treatment for 30 months. Subjects were maintained on Gattex even if no longer requiring PN/I.V. support. These 10 subjects had required PN/I.V. support for 1.2 to 15.5 years, and prior to Gattex had required between 3.5 L/week and 13.4 L/week of PN/I.V. support. At the end of study, 21 (70%), 18 (60%) and 18 (60%) of the 30 completers achieved a reduction of 1, 2, or 3 days per week in PN/I.V. support, respectively.

24 month exposure

Of the 39 placebo subjects from STEPS entering STEPS 2, 29 completed 24 months of treatment with Gattex. The mean reduction in PN/I.V. was 3.11 L/week (an additional 28.3% reduction) from the start of STEPS 2. Sixteen (55.2%) of the 29 completers achieved a 20% or greater reduction of parenteral support. At the end of study, 14 (48.3%), 7 (24.1%) and 5 (17.2%) achieved a reduction of 1, 2, or 3 days per week in PN/I.V. support, respectively. Two subjects were weaned off their PN/I.V. support while on Gattex. Of the 12 subjects entering STEPS 2 directly, 6 completed 24 months of treatment with Gattex. Similar effects were seen. One of the six subjects was weaned off their PN/I.V. support while on Gattex.

The rates of adverse events of special interest as described in the U.S. Prescribing Information remain consistent. The most common adverse events in STEPS 2 were abdominal pain, catheter sepsis, and episodes of decreased weight, nausea, gastrointestinal stoma complications, and abdominal distension.

About Gattex® (Teduglutide [rDNA origin]) for Injection

Gattex® (teduglutide [rDNA origin]) for injection for subcutaneous use is a novel, recombinant analog of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. Gattex is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. Significant reductions in mean PN/IV infusion volume from baseline to end of treatment were seen in the Phase 3 studies of Gattex. In addition, some patients were able to achieve independence from PN/IV support during these trials. The most common side effects of Gattex include stomach area (abdomen) pain or swelling, skin reaction where the injection was given, nausea, headache, cold or flu like symptoms, vomiting, and holding too much fluid in the body (swelling of face, ankles, hands or feet).

The European Commission granted European market authorization on August 30, 2012 for the medicinal product teduglutide (trade name in Europe: Revestive®) as a once-daily treatment for adult patients with SBS.

Teduglutide has orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and the FDA.

Important Safety Information

What is the most important information I should know about GATTEX?

GATTEX may cause serious side effects, including:

Making abnormal cells grow faster

GATTEX can make abnormal cells that are already in your body grow faster. There is an increased risk that abnormal cells could become cancer. If you get cancer of the bowel (intestines), liver, gallbladder or pancreas while using GATTEX, your healthcare provider should stop GATTEX. If you get other types of cancers, you and your healthcare provider should discuss the risks and benefits of using GATTEX.

Polyps in the colon (large intestine)

Polyps are growths on the inside of the colon. Your healthcare provider will have your colon checked for polyps within 6 months before starting GATTEX and have any polyps removed.

To keep using GATTEX, your healthcare provider should have your colon checked for new polyps at the end of 1 year of using GATTEX. If no polyp is found, your healthcare provider should check you for polyps as needed and at least every 5 years and have any new polyps removed. If cancer is found in a polyp, your healthcare provider should stop GATTEX.

Blockage of the bowel (intestines)

A bowel blockage keeps food, fluids, and gas from moving through the bowels in the normal way. Tell your healthcare provider if you have any of these symptoms of a bowel blockage:

• trouble having a bowel movement or passing gas
• stomach area (abdomen) pain or swelling
• nausea
• vomiting
• swelling and blockage of your stoma opening, if you have a stoma

If blockage is found, your healthcare provider may temporarily stop GATTEX.

Swelling (inflammation) or blockage of your gallbladder or pancreas

Your healthcare provider will do tests to check your gallbladder and pancreas within 6 months before starting GATTEX and at least every 6 months while you are using GATTEX. Tell your healthcare provider right away if you get stomach area (abdomen) pain and tenderness, chills, fever, change in your stools, nausea, vomiting, dark urine, or yellowing of your skin or the whites of eyes.

Fluid overload

Your healthcare provider will check you for too much fluid in your body. Too much fluid in your body may lead to heart failure, especially if you have heart problems. Tell your healthcare provider if you get swelling in your feet and ankles, you gain weight very quickly (water weight), or you have trouble breathing.

The most common side effects of GATTEX include:

• stomach area (abdomen) pain or swelling
• skin reaction where the injection was given
• nausea
• headache
• cold or flulike symptoms
• vomiting

Tell your healthcare provider if you have any side effect that bothers you or that does not go away.

What should I tell my healthcare provider before using GATTEX?

Tell your healthcare provider if you:

• Have cancer or a history of cancer
• Have or had polyps anywhere in your bowel (intestines) or rectum
• Have heart problems
• Have high blood pressure
• Have problems with your gallbladder, pancreas, kidneys
• Have any other medical condition
• Are pregnant or planning to become pregnant. It is not known if GATTEX will harm your unborn baby. Tell your healthcare provider right away if you become pregnant while using GATTEX.
• Are breastfeeding or plan to breastfeed. It is not known if GATTEX passes into your breast milk. You and your healthcare provider should decide if you will use GATTEX or breastfeed. You should not do both.

Tell your healthcare providers about all the medicines you take, including prescription or over-the-counter medicines, vitamins, and herbal supplements. Using GATTEX with certain other medicines may affect each other causing side effects. Your other healthcare providers may need to change the dose of any oral medicines you take while using GATTEX. Tell the healthcare provider who gives you GATTEX if you will be taking a new oral medicine.

Call your doctor for medical advice about side effects. To report suspected side effects, contact NPS Pharma at 1-855-5GATTEX (1-855-542-8839) or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

About NPS Pharma

NPS Pharma is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company's lead product, Gattex® (teduglutide [rDNA origin]) for injection is approved in the US for adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. In the EU, teduglutide (trade name: Revestive®) is approved for the treatment of adult patients with SBS; patients should be stable following a period of intestinal adaptation after surgery. Teduglutide is not approved for the treatment of pediatric SBS patients. The safety and efficacy of teduglutide in this population is currently being evaluated in a global registration trial.

A Biologics License Application is undergoing FDA review for Natpara® (rhPTH [1-84]) for the treatment of hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone. The Prescription Drug User Fee Act goal date for the Natpara application is October 24, 2014.

NPS Pharma's earlier stage pipeline includes NPSP795, a calcilytic compound with potential application in rare disorders involving increased calcium sensing receptor activity, such as autosomal dominant hypocalcemia (ADH). NPS Pharma complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin.

“NPS Pharma,” “NPS Pharmaceuticals,” “Gattex,” and “Revestive” are the company's trademarks.

Disclosure notice

Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements are based on the company's current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Forward looking statements include, but are not limited to, statements concerning the company's future financial performance and plans for the commercialization of its products, beliefs or expectations regarding our products in development, statements concerning the company's plans for international expansion, beliefs or expectations regarding potential revenue and earnings from product sales, including beliefs regarding our ability to grow sales, expectations regarding the market size for our products, including those in development, and beliefs or expectations regarding our operating expenses. Risks associated to the company's business include, but are not limited to, the risks associated with any failure by the company to successfully commercialize Gattex/Revestive (teduglutide [rDNA origin]) for injection, including the risk that physicians and patients may not see the advantages of Gattex/Revestive and may therefore be reluctant to utilize the product, the risk that private and public payers may be reluctant to cover or provide reimbursement for Gattex, risks related to regulatory approvals for recombinant human parathyroid hormone 1-84 (rhPTH [1-84]), the risks associated with the company's strategy, global macroeconomic conditions, the impact of changes in management or staff levels, the effect of legislation effecting healthcare reform in the United States, as well as other risk factors described in the company's periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Form 10-Qs. All information in this press release is as of the date of this press release and NPS undertakes no duty to update this information, whether as a result of new information, future events or otherwise.