Posted by: ctraderd | July 26, 2012

FDA Approvals- 7/26/2012

On July 26, 2012, The FDA had two PDUFAs set. The PDUFAs were for the companies Horizon Pharmaceuticals (HZNP) and their drug NP01, and Amarin Corp, PLC (AMRN) and their drug AMR101. Both drugs have been approved, but only HZNP has allowed trading after approval.

HZNP was halted during the open market due to approval, but did not open trading until 4:40 P.M. EST, when the market was closed. In afterhours trading, HZNP spiked to 8.20, then fell down to a low of approximately 6.10. The stock then rebounded to the price of 7.20, then fell again to 6.75, which is where it closed the aftermarket trading at. HZNP closed the day out losing –.87 for total of –11.42%. In the last part of the day, the shorts painted the tape. HZNP will market NP01 under the name RAYOS, which is a treatment for rheumatoid arthritis.

FDA Press Release: 

DEERFIELD, Ill., July 26, 2012 /PRNewswire/ — Horizon Pharma, Inc., (NASDAQ: HZNP) announced today that the U.S. Food and Drug Administration (FDA) has approved RAYOS® (prednisone) delayed-release tablets (1 mg, 2 mg and 5 mg) to treat a broad range of diseases including rheumatoid arthritis (RA), polymyalgia rheumatica (PMR), psoriatic arthritis (PsA), ankylosing spondylitis (AS), asthma and chronic obstructive pulmonary disease (COPD) (see full prescribing information at www.RAYOSrx.com). The FDA approval was supported by data bridging the pharmacokinetics of RAYOS to immediate-release prednisone and data from the Circadian Administration of Prednisone in RA (CAPRA-1 and 2) trials. The CAPRA-2 trial demonstrated that people with moderate to severe RA treated with RAYOS experienced a statistically significant improvement in ACR20 response criteria compared to placebo. The CAPRA-1 trial supported the overall safety of RAYOS.

"We are extremely pleased the FDA has approved RAYOS for a broad range of indications, including RA and polymyalgia rheumatica," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma. "Our initial focus will be on the launch of RAYOS in rheumatologic diseases such as RA and polymyalgia rheumatica in the fourth quarter of this year. Based on the extent of the approved indications, we will be developing a broader commercial strategy to expand the opportunity for RAYOS in key IL-6 mediated diseases, including asthma and COPD."

RAYOS Clinical Data
U.S. New Drug Application
The efficacy of RAYOS in the treatment of RA was assessed in the CAPRA-2 trial, a double-blind, placebo-controlled, randomized, 12-week trial in patients with active rheumatoid arthritis diagnosed according to American College of Rheumatology (ACR) criteria. Enrolled patients were not currently being treated with corticosteroids but had received non-biologic disease-modifying antirheumatic drug (DMARD) therapy for at least 6 months prior to receipt of study medication, with an incomplete response to DMARD therapy alone. Patients were randomized in a 2:1 ratio to treatment with RAYOS 5 mg (n=231) or placebo (n=119) administered at 10 p.m. in addition to their DMARD therapy. A total of 350 patients were enrolled and ranged in age from 27 to 80 years (median age 57 years). Patients were predominantly Caucasian and 84% were female.

Results from CAPRA-2 demonstrated:

  • A statistically significant improvement in ACR20 response criteria, the primary study endpoint, for patients who were treated with RAYOS compared to the placebo group (47% vs. 29%; p-value = 0.001).
  • A statistically significant improvement in ACR50 response compared to placebo (22% vs. 10%; p-value = 0.007) and an improvement in the more stringent ACR70 response criteria (7% vs. 3%; p-value = 0.0984). Both ACR50 and ACR70 were pre-specified secondary endpoints.

The relative change from baseline in the duration of morning stiffness at 12 weeks was assessed as a pre-specified secondary endpoint. Patients treated with RAYOS had a median decrease in the duration of morning stiffness of 55 minutes compared to 33 minutes in placebo-treated patients (20 minute estimated median difference between treatment groups with 95% confidence interval [7, 32; p-value = 0.001]).

Results from CAPRA-2 are published in Annals of the Rheumatic Diseases.1

"Prednisone is a common therapy for patients with various inflammatory diseases, including RA, and the delayed-release enhancement offered with RAYOS is an important treatment advance," said Michael Schiff, M.D., Clinical Professor of Medicine at the University of Colorado School of Medicine, Rheumatology Division. "RAYOS is engineered to benefit the underlying patterns of inflammatory diseases. RAYOS, as studied in its clinical trials with ten p.m. dosing, reduces the overnight rise of inflammatory mediators, which results in less pain and stiffness for patients as they begin their day."

The safety of RAYOS was based on the evaluation of 375 RA patients in two controlled trials. Patients treated with RAYOS ranged in age from 20 to 80 years (median age 56 years). Patients were predominantly Caucasian and 85% were female.

Included in these safety results were data from the CAPRA-1 trial, a 12 week, double-blind, placebo-controlled study that evaluated 288 RA patients. CAPRA-1 compared 10 p.m. administration of RAYOS with the morning administration of immediate-release prednisone at the same individual dose (average dose of 6.7 mg). Following the 12-week CAPRA-1 study, patients were followed in a 9-month, open-label extension study, which included 249 RA patients, 219 of whom completed the extension study. Patients received RAYOS 3 mg to 10 mg once daily at 10 p.m.; the majority (84%) received 5 mg or less.

The clinical trial experience did not raise any safety concerns beyond those already established for immediate-release prednisone.

Results from the CAPRA-1 12-week study and the 9-month open-label extension are published in The Lancet and Annals of the Rheumatic Diseases, respectively.2,3

About RAYOS
RAYOS, known as LODOTRA® in Europe, is a proprietary delayed-release formulation of low-dose prednisone. The pharmacokinetic profile of RAYOS is different with an approximately four-hour lag time from that of immediate-release prednisone formulations. In clinical trials studying use of RAYOS in RA, patients were administered RAYOS at 10 p.m. with food. Given RAYOS delayed-release profile, this helps to achieve therapeutic prednisone blood levels at a time point when cytokine levels start rising during the middle of the night. While the pharmacokinetic profile of RAYOS differs in terms of lag time from immediate-release prednisone, its absorption, distribution and elimination processes are comparable

RAYOS utilizes SkyePharma’s proprietary Geoclock™ technology.Outside the United States, LODOTRA is approved for the treatment of moderate to severe active RA when accompanied by morning stiffness in 16 European countries and Israel. Horizon has granted commercialization rights for LODOTRA in Europe, Asia and Latin America to its distribution partner Mundipharma International Corporation Limited.

Important Safety Information

RAYOS® (prednisone) delayedrelease tablets

Approved uses of RAYOS

RAYOS, a delayed‐release form of prednisone, prevents the release of substances in the body that cause inflammation. RAYOS is approved to treat a broad range of diseases including rheumatoid arthritis (RA), polymyalgia rheumatica (PMR), psoriatic arthritis (PsA), ankylosing spondylitis (AS), asthma and chronic obstructive pulmonary disease (COPD). For a full list of RAYOS indications, please see full prescribing information at www.RAYOSrx.com.

RAYOS is contraindicated in patients who have known hypersensitivity to prednisone or to any of the excipients. Rare instances of anaphylaxis have occurred in patients receiving corticosteroids.

Important information about RAYOS
Do not use RAYOS if you are allergic to prednisone.

Long‐term use of RAYOS can affect how your body responds to stress. Symptoms can include weight gain, severe fatigue, weak muscles, and high blood sugar.

RAYOS can weaken your immune system, making it easier for you to get an infection or worsening an infection you already have or have recently had.

RAYOS can cause high blood pressure, salt and water retention and low blood potassium.

There is an increased risk of developing holes in the stomach or intestines if you have certain stomach and intestinal disorders.

Behavior and mood changes can occur, including intense excitement or happiness, sleeplessness, mood swings, personality changes or severe depression.

Long‐term use of RAYOS can cause decreases in bone density.

RAYOS can cause cataracts, eye infections and glaucoma.

Do not receive a "live" vaccine while taking RAYOS. The vaccine may not work as well during this time, and may not fully protect you from disease.

Taking RAYOS during the first trimester of pregnancy can harm an unborn baby.

Long‐term use of RAYOS can slow growth and development in children.

The most common side effects with RAYOS are water retention, high blood sugar, high blood pressure, unusual behavior and mood changes, increased appetite and weight gain.

Please see full prescribing information for RAYOS at www.RAYOSrx.com.

About Horizon Pharma
Horizon Pharma, Inc. (NASDAQ: HZNP) is a biopharmaceutical company that is developing and commercializing innovative medicines to target unmet therapeutic needs in arthritis, pain and inflammatory diseases. For more information, please visit www.horizonpharma.com.

Forward Looking Statements
This press release contains forward-looking statements, including statements regarding the timing of a potential commercial launch of RAYOS in the United States, the company’s plans to develop a broader commercial strategy for RAYOS and the potential for RAYOS to provide a new treatment option for patients with inflammatory diseases. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors, including, but not limited to, risks regarding the company’s ability to commercialize products successfully, changes in the company’s strategy as to when to launch RAYOS in the United States and on which approved indications it will focus its initial commercial efforts, whether physicians will prescribe and patients will use RAYOS, once available, and competition in the market for RAYOS. For a further description of these and other risks facing the company, please see the risk factors described in the company’s filings with the United States Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in those filings. Forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to update or revise these statements, except as may be required by law.

Source: http://finance.yahoo.com/news/horizon-pharma-announces-fda-approval-201000748.html

HZNP(3)

AMRN was halted before HZNP, but HZNP got approval before AMRN, but AMRN did not allow afterhours trading and closed the day gaining +.73 for a total of +5.04%. AMRN will market AMR101 under the name Vascepa, which is a pill to reduce high triglyceride levels.

FDA Press Release:

BEDMINSTER, N.J. and DUBLIN, Ireland, July 26, 2012 (GLOBE NEWSWIRE) — Amarin Corporation plc (Nasdaq:AMRN), a biopharmaceutical company focused on the commercialization and development of therapeutics to improve cardiovascular health, announced today that the U.S. Food and Drug Administration (FDA) has approved VascepaTM (icosapent ethyl) capsules (formerly known as AMR101) as an adjunct to diet to reduce triglyceride (TG) levels in adult patients with severe (TG greater than or equal to 500mg/dL) hypertriglyceridemia (very high triglycerides). Amarin submitted the New Drug Application (NDA) for the use of Vascepa in this indication in September 2011.

"FDA approval of Vascepa represents the introduction of an important new treatment option for patients with severe hypertriglyceridemia. In Amarin’s MARINE clinical trial, Vascepa demonstrated a statistically significant placebo-adjusted reduction in levels of triglycerides without elevation in levels of LDL-C, commonly referred to as ‘bad cholesterol,’" stated Joseph Zakrzewski, Chairman and CEO of Amarin. "Amarin continues to anticipate commercial launch of Vascepa early in the first quarter of 2013, and we continue to consider three potential paths for the marketing and sale of the product: an acquisition of Amarin, a strategic collaboration, or self-commercialization, the latter of which could include third-party support. We are now focused on continued commercial preparations for Vascepa which includes, but is not limited to, finalizing the introduction of Vascepa to managed care plans to gain formulary access, building-up inventory levels and coordinating other pre-launch marketing activities."

The efficacy and safety of Vascepa were assessed in Amarin’s MARINE clinical trial, a randomized, placebo-controlled, double-blind, parallel-group study of adult patients with very high fasting triglyceride levels, between 500 mg/dL and 2000 mg/dL. At baseline, 25% of patients were on concomitant statin therapy, 28% were diabetics, and 39% of patients had TG levels greater than 750 mg/dL. Patients treated for 12 weeks with the 4 gram dose of Vascepa demonstrated a statistically significant placebo-adjusted median triglyceride reduction of 33% (p < 0.001), and did not show an increase in LDL-C levels relative to placebo. Vascepa 4 grams per day also showed statistically significant placebo-adjusted median reductions from baseline in non-HDL-C (total cholesterol less "good cholesterol") of 18%, Total Cholesterol (TC) of 16%, Very Low Density Lipoprotein Cholesterol (VLDL-C) of 29%, and apolipoprotein B (Apo B) of 9%.

The most commonly reported adverse reaction (incidence > 2% and greater than placebo) in Vascepa treated patients was arthralgia (joint pain) (2.3% for Vascepa vs. 1.0% for placebo).

The following table shows the changes in major lipoprotein and lipid parameters for the treatment groups:

Median Baseline and Percent Change from Baseline in Lipid Parameters in Patients with Severe (greater than or equal to 500 mg/dL) Hypertriglyceridemia


Vascepa 4 g/day
Placebo


N=76
N=75

Parameter
Baseline
% Change
Baseline
% Change
Difference (95%
Confidence
Interval)

TG (mg/dL)
680
-27
703
+10
-33* (-47, -22)

LDL-C (mg/dL)
91
-5
86
-3
-2 (-13, +8)

Non-HDL-C (mg/dL)
225
-8
229
+8
-18 (-25, -11)

TC (mg/dL)
254
-7
256
+8
-16 (-22, -11)

HDL-C (mg/dL)
27
-4
27
0
-4 (-9, +2)

VLDL-C (mg/dL)
123
-20
124
+14
-29** (-43, -14)

Apo B (mg/dL)
121
-4
118
+4
-9**(-14, -3)

% Change= Median Percent Change from Baseline

Difference= Median of [Vascepa % Change — Placebo % Change] (Hodges-Lehmann Estimate)

p-values from Wilcoxon rank-sum test

*p-value < 0.001 (primary efficacy endpoint)

**p-value < 0.05 (key secondary efficacy endpoints determined to be statistically significant according to the pre-specified multiple comparison procedure)

Apo B = apolipoprotein B; HDL-C = high-density lipoprotein cholesterol; LDL-C = low-density lipoprotein cholesterol; non-HDL-C = non-high-density lipoprotein cholesterol; TC = total cholesterol; TG = triglyceride; VLDL-C = very-low-density lipoprotein cholesterol

Note: All endpoints in the above table for MARINE were statistically significant with the exception of HDL-C.

Amarin has made significant progress in its efforts to expand the patent protection for Vascepa in the United States to at least 2030 with seven patent applications either issued, allowed or in progressed states of prosecution and over 25 additional U.S. applications pending. Amarin is awaiting a decision from FDA as to whether Vascepa will be granted five-year new chemical entity (NCE) or three-year new product marketing exclusivity under the provisions of the Hatch-Waxman amendments to the Federal Food, Drug, and Cosmetic Act. Typically, FDA’s determination on the exclusivity of approved products is made public through the posting on FDA’s website in the Orange Book. This typically occurs mid-month following the month of an NDA approval.

About VascepaTM (icosapent ethyl) capsules

VascepaTM (icosapent ethyl) capsules, known in scientific literature as AMR101, is a patented, ultra-pure omega-3 fatty acid product, comprising not less than 96% EPA in a 1 gram capsule.

IMPORTANT PRESCRIBING INFORMATION

Vascepa™ (icosapent ethyl) is indicated for use in the United States as an adjunct to diet to reduce triglyceride (TG) levels in adult patients with severe (greater than or equal to500 mg/dL) hypertriglyceridemia.

The effect of Vascepa on the risk for pancreatitis in patients with severe hypertriglyceridemia has not been determined.

The effect of Vascepa on cardiovascular mortality and morbidity in patients with severe hypertriglyceridemia levels has not been determined.

The daily dose of Vascepa is 4 grams administered orally. Patients should engage in appropriate nutritional intake and physical activity before receiving Vascepa, which should continue during treatment.

FULL VASCEPA PRESCRIBING INFORMATION CAN BE FOUND AT WWW.VASCEPA.COM

IMPORTANT SAFETY INFORMATION

Attempts should be made to control any medical problems such as diabetes mellitus, hypothyroidism and alcohol intake that may contribute to the lipid abnormalities. Lipid levels should be consistently abnormal before initiating Vascepa.

Medications affecting coagulation (e.g., anti-platelet agents) should be monitored periodically.

In patients with hepatic impairment, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels should be monitored periodically during therapy. Use with caution in patients with known hypersensitivity to fish and/or shellfish.

The most commonly reported adverse reaction (incidence > 2% and greater than placebo) in Vascepa treated patients was arthralgia (joint pain) (2.3% for Vascepa vs. 1.0% for placebo).

About Severe (greater than or equal to 500 mg/dL) Hypertriglyceridemia

Severe hypertriglyceridemia refers to a condition in which patients have very high levels of triglycerides (greater than or equal to 500 mg/dL) in the bloodstream. Amarin estimates that approximately 4 million people in the United States have severe hypertriglyceridemia. According to The American Heart Association Scientific Statement on Triglycerides and Cardiovascular Disease (2011), triglycerides provide important information as a marker associated with the risk for heart disease and stroke, especially when an individual also has low high-density lipoprotein cholesterol, or HDL-C (often referred to as "good" cholesterol), and elevated levels of LDL-C (often referred to as "bad" cholesterol). The effect of Vascepa on cardiovascular mortality and morbidity in patients with severe hypertriglyceridemia has not been determined.

Conference call for investors

Amarin will host a conference call and webcast for investors, today at 7:00 p.m. EDT to discuss FDA’s approval of Vascepa. The conference call will be webcast live and a link to the webcast may be accessed from the "Events & Presentations" page on the Amarin corporate website at www.amarincorp.com.

To listen to the live call on the telephone, dial 1-877-407-8033 (United States and Canada) or 1-201-689-8033 (International). The conference call ID number is 397905. A replay of the call will be available for 30 days by dialing 1-877-660-6853 (United States and Canada) or 1-201-612-7415 (International), account number 286, conference ID 397905.

About Amarin

Amarin Corporation plc is a biopharmaceutical company focused on the commercialization and development of therapeutics to improve cardiovascular health. Amarin’s product development program leverages its extensive experience in lipid science and the potential therapeutic benefits of polyunsaturated fatty acids. VascepaTM (icosapent ethyl) is Amarin’s first FDA approved product. For more information about Vascepa visit www.vascepa.com. Amarin plans to separately seek approval for the use of Vascepa in the treatment of patients with high triglyceride levels who are also on statin therapy for elevated LDL-C levels, the population studied in Amarin’s ANCHOR trial, after Amarin’s REDUCE-IT cardiovascular outcomes trial is substantially underway. Like Amarin’s MARINE study, each of Amarin’s ANCHOR and REDUCE-IT studies is the subject of a Special Protocol Assessment (SPA) agreement with the FDA. For more information about Amarin visit www.amarincorp.com.

The Amarin Corporation plc logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13817

Forward-looking statements

This press release contains forward-looking statements, including statements about the efficacy, safety and therapeutic benefits of Vascepa, clinical trial results, the clinical importance of certain biomarkers and the impact of Vascepa on such biomarkers, the timing of a commercial launch of Vascepa, the potential additional indications for which FDA marketing approval of Vascepa may be sought and the commercial potential of Vascepa, the potential for an acquisition of Amarin or a strategic collaboration with a third party for the commercialization of Vascepa, the timing and outcome of FDA’s review determination of whether Vascepa should be granted new chemical entity or new product marketing exclusivity, the status of patent applications currently under review by the United States Patent and Trademark Office, the coverage and expected expiration dates of those patent applications and issued patents and the ability of Amarin to protect the commercial potential of Vascepa. In particular there can be no assurance that Vascepa will be awarded five-year new chemical entity or three-year new product marketing exclusivity and the FDA may take longer than expected to reach any such determination. Amarin’s patent portfolio directed to the formulation and uses of Vascepa is still evolving and some patent applications may not issue prior to commercial launch, if ever. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include the following: uncertainties associated generally with the commercial success of new pharmaceutical products, such as Vascepa; Amarin’s ability to negotiate and execute a successful acquisition of Amarin or a strategic collaboration with a third party for the commercialization of Vascepa; Amarin’s lack of experience with commercializing pharmaceutical products; risks associated with preparations associated with a commercial launch; the risk that FDA may not grant new chemical entity or new product marketing exclusivity to Vascepa; the risk that FDA may not reach a determination with respect to these matters on the timetable that we expect; the risk that patent applications may not result in issued patents, and that issued patents may not prevent competitors from competing with Vascepa; the risk that competitors may challenge the validity, enforceability or both the validity and enforceability of our patents or seek to design products around our issued patent claims and gain marketing approval for generic versions of Vascepa or branded competitive products based on new clinical studies; and the risk that trade secrets may not be maintained and that circumstances that create manufacturing barriers to entry may not last. A further list and description of these risks, uncertainties and other risks associated with an investment in Amarin can be found in the "Risk Factors" section of Amarin’s filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Amarin undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise except as required by law.

Amarin’s product candidates are in various stages of development and are not available for sale or use outside of approved clinical trials, except as it relates to the FDA approval announced herein. This press release is intended for communication with investors. Nothing in this press release should be construed as marketing the use of such product candidates.

Source: http://investor.amarincorp.com/releasedetail.cfm?ReleaseID=696027

AMRN(2)

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