surf1944
11 years ago
2:36AM Santarus and Pharming announce new data from open-label repeat treatment study with RUCONEST (SNTS) 23.22 : Santarus and Pharming Group NV announce that new data from an open-label extension of the pivotal Phase III clinical study with RUCONEST. The median time in minutes to onset of symptom relief following treatment as measured by patient responses to a Treatment Effect Questionnaire for the first five repeat attacks ranged from 62.5 (48, 90) to 134.0 (75, 150) and across all attacks was a median of 75.0 (69, 89). The median (95% CI) time in minutes to minimal symptoms (first three attacks per patient) as measured by a TEQ ranged from 243 (76, 1440) to 304 (150, 719) and for all assessed attacks was 303 (211, 367).
Exposure and Safety Information
Only one dose of RUCONEST was administered for 96% of the 224 attacks.
12 of 44 (27%) patients experienced at least one treatment emergent adverse event within 72 hours of completion of RUCONEST infusion.
TEAEs occurring in 5% of patients were nasopharyngitis, cough, fibrin D-dimer increase, and headache
The percentage of patients experiencing TEAEs did not increase with RUCONEST treatments for repeat attacks.
There were no discontinuations due to adverse events, no thrombotic or anaphylactic events, and no neutralizing anti-C1INH antibodies observed with repeat RUCONEST treatment.
Santarus and Pharming are seeking U.S. marketing approval of RUCONEST for the treatment of acute angioedema attacks in patients with HAE. The Biologics License Application filing for RUCONEST is under review by the FDA with a response expected by April 16, 2014.
surf1944
11 years ago
7:01AM Santarus announces presentation of SAN-300 Phase I data at 2013 American College of Rheumatology Annual Meeting; plan to initiate Phase IIa clinical study in patients with rheumatoid arthritis by year-end 2013 (SNTS) 24.60 : Co announced that data from its Phase I clinical study with its investigational drug SAN-300, a novel antibody that inhibits very late antigen-1 (VLA-1; a1 1 integrin), will be featured in a poster presentation at the 2013 American College of Rheumatology. Based on the Phase I study results, Santarus is preparing to initiate a multicenter, randomized, placebo-controlled, multiple ascending dose, Phase IIa clinical study with subcutaneous administration of SAN-300 in patients with active rheumatoid arthritis. The co expects to initiate the Phase IIa study in the fourth quarter of 2013.
surf1944
11 years ago
7:04AM Santarus announces presentation of Phase III data for Rifamycin SV MMX in Travelers' Diarrhea (SNTS) 22.58 : Co announced that new data from its Phase III clinical study with the investigational drug rifamycin SV MMX in patients with travelers' diarrhea will be featured in a poster presentation at IDWeek in San Francisco at the Moscone Center (Poster Hall C) from 12:30 p.m. to 2:00 p.m. Pacific Time on Saturday, Oct 5, 2013. IDWeek is the combined annual meeting of the Infectious Diseases Society of America (IDSA), the Society for Healthcare Epidemiology of America (SHEA), the HIV Medicine Association (HIVMA), and the Pediatric Infectious Diseases Society (PIDS).
"The data from this clinical study indicate that rifamycin SV MMX significantly shortened the duration of travelers' diarrhea and was well tolerated, with adverse events similar to that of placebo in the study. In addition, rifamycin SV MMX demonstrated broad activity, and of interest was the possible activity against invasive pathogens," said Herbert L. DuPont, M.D., principal investigator and lead author of the poster. Dr. DuPont is Director of the Center for Infectious Diseases and Professor of Infectious Diseases at the University of Texas -- Houston School of Public Health, Chief of Internal Medicine at St. Luke's Hospital and Vice Chairman, Department of Medicine and Clinical Professor at the Baylor College of Medicine.
The co has previously announced that the Phase III study met the primary endpoint of reducing the time to last unformed stool (TLUS) in the intent-to-treat (ITT) population.
Zarathos
11 years ago
Uceris sucks, plain and simple. If anyone reading this has ulcerative colitis, DO NOT substitute Uceris for prednisone. I just tried that and it nearly put me in the hospital. Furthermore, I have an acquaintance who also has UC. In an unbelievable coincidence, he is following nearly identical to me right now, except he waited to switch to prednisone a few days longer than I did and he is still in agony. I own SNTS, but I'm so outraged that I am posting this now, even though I still own it. I am so pissed that I will probably sell my SNTS Monday. If you are a UC patient and reading this, avoid Uceris.
surf1944
11 years ago
7:05AM Santarus and VeroScience announce publication of AACE expert panel recommendations for CYCLOSET; Experts conclude that favorable cardiovascular profile of CYCLOSET suggests it may be useful in the treatment of patients with type 2 diabetes with significant risk factors or history of cardiovascular disease (SNTS) 15.33 : Co announced publication of a review article and recommendations of an American Association of Clinical Endocrinologists (AACE) expert panel on the role of bromocriptine-QR in the management of type 2 diabetes. The panel concluded that treatment with CYCLOSET (bromocriptine mesylate), also referred to as bromocriptine-QR, may be useful in the treatment of patients with type 2 diabetes, both early and late in the life cycle of the disease, and especially for patients with a history of cardiovascular disease or who have significant risk factors for cardiovascular disease.
Adjunctive administration of CYCLOSET in the dosing range of 1.6 mg/day to 4.8 mg/day may result in a mean reduction in A1c levels of 0.69%. A 24-week completer analysis performed among efficacy subgroups of patients from the CYCLOSET safety study stratified based on the patient's baseline A1c levels yielded the following results: in the subgroup treated with metformin, treatment with bromocriptine-QR was associated with a significantly higher proportion of subjects achieving a Week 24 A1c level =7 compared with placebo when baseline A1c levels were =7.5 to <8.0 or 8.0 to 8.5.
The large (n=3,070) placebo-controlled 12-month safety study provides the most detailed and systematic information on the long-term tolerability of CYCLOSET in the treatment of type 2 diabetes. Nausea was the most common adverse event (32.2%), and was associated with premature study discontinuation in 7.6% of patients
surf1944
12 years ago
7:01AM Santarus and VeroScience announce publication of 'positive' data from pivotal CYCLOSET (bromocriptine mesylate) efficacy study; CYCLOSET add-on therapy provided significant improvement in glycemic control versus placebo in patients with type 2 diabetes poorly controlled on one or two oral anti-diabetes medications (SNTS) 12.37 : Co and VeroScience announced publication of positive data from a double-blind, placebo-controlled, multi-center pivotal study with CYCLOSET (bromocriptine mesylate) tablets. Patient groups in the evaluable per protocol (EPP) population that added CYCLOSET to their treatment regimen achieved a 0.60% to 0.70% reduction in HbA1c (p<0.0001) relative to placebo after 24 weeks, the primary endpoint of the study. These results appear in the Nov/Dec 2012 print edition of Endocrine Practice, a peer-reviewed medical journal. Additionally, among the EPP population with no concomitant change in diabetes medication, treatment with CYCLOSET resulted in reductions in HbA1c levels of 0.69% to 0.83% (CYCLOSET vs. placebo between group difference, p<0.0001) as well as five to seven times more subjects reaching the goal of HbA1c =7.0 in the CYCLOSET treated groups compared with placebo (32%-42% vs 5%-7%, respectively) (p<0.0001).
surf1944
12 years ago
1:34AM Santarus sees FY12 and FY13 revs above consensus; co also announced it received FDA approval of UCERIS (SNTS) 11.28 : Co issues upside guidance for FY12 (Dec), sees FY12 (Dec) revs of ~$210 mln vs. $209.33 mln Capital IQ Consensus Estimate. Co issues upside guidance for FY13 (Dec), sees FY13 (Dec) revs of ~$320-325 mln vs. $301.25 mln Capital IQ Consensus Estimate. Financial Outlook for 2012 and 2013 For the full year in 2012, co expects to meet or exceed the financial guidance provided on its November 7, 2012 third quarter financial results conference call of:
Net income of ~$12-14 mln, which includes the impact of the ~$4 mln success-based regulatory milestone expense in the first quarter of 2012 related to UCERIS and a $10 mln expense for the success-based clinical milestone for RUCONEST in the fourth quarter.
Adjusted EBITDA of ~$29-32 mln.
Co is introducing its financial outlook for full year 2013 as follows:
Net income of approximately $50-54 mln.
Adjusted EBITDA of ~$73-79 mln.
*Co also announced it received FDA approval of UCERIS for the induction of remission in patients with active, mild to moderate ulcerative colitis.
1:30AM Santarus receives FDA approval of UCERIS for the induction of remission in patients with active, mild to moderate ulcerative colitis (SNTS) 11.28 : Co announces the FDA has approved UCERIS extended release tablets for the induction of remission in patients with active, mild to moderate ulcerative colitis. The co expects to commence the commercial launch of UCERIS in March 2013.
surf1944
12 years ago
7:03AM Santarus announced publication of new analysis of CYCLOSET; CYCLOSET demonstrated a significant 52% relative risk reduction in cardiovascular events (SNTS) 9.03 : Co disclosed 52-week, randomized safety study with CYCLOSET tablets were published in the Journal of the American Heart Association, an online publication. CYCLOSET is a unique, quick release form of bromocriptine, a dopamine D2 receptor agonist, and is approved as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus. In this safety study, a total of 3,070 patients on stable doses of up to two antidiabetes medications with HbA1c = 10.0 (average baseline HbA1c=7.0) were randomized 2:1 to CYCLOSET or placebo for a 52-week treatment period. Patients with heart failure (New York Heart Classes I and II) and precedent myocardial infarction or revascularization surgery were allowed to participate in the trial.
Regarding the MACE endpoint, there were 14 events (0.7%) among 2,054 CYCLOSET-treated subjects and 15 events (1.5%) among 1,106 placebo-treated subjects, yielding a significant, 52% reduction in relative risk in cumulative percent of cardiovascular events over time of this MACE endpoint. With respect to the cardiovascular death-inclusive cardiovascular endpoint, there were 39 events (1.9%) among 2,054 CYCLOSET-treated subjects versus 33 events (3.2%) among 1,016 placebo subjects, yielding a significant, 39% reduction in relative risk in the cumulative percent over time of this composite cardiovascular endpoint.
"Our analysis indicated that bromocriptine-QR, or CYCLOSET, significantly reduced the cardiovascular death-inclusive composite cardiovascular endpoint by 39% in type 2 diabetes patients after one year of treatment. Importantly, this observed relative risk reduction was consistent regardless of age, race, sex, duration of disease or preexisting macrovascular disease...Furthermore, among patients in the study, CYCLOSET significantly reduced the relative risk for the composite cardiovascular endpoint of myocardial infarction, stroke and cardiovascular death by 52%."
surf1944
12 years ago
1:28AM Santarus and Pharming announce positive top-line Phase III results for RUCONEST in acute hereditary angioedema (SNTS) 9.17 : Co and Pharming Group NV announce that their pivotal Phase III clinical study to evaluate the safety and efficacy of the investigational drug RUCONEST 50 U/kg for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema met the primary endpoint of time to beginning of symptom relief. A statistically significant difference in the time to beginning of symptom relief was observed in the intent-to-treat population between RUCONEST and placebo; the median time to beginning of symptom relief was 90 minutes for RUCONEST patients and 152 minutes for placebo patients. The time to beginning of symptom relief was defined as the time from the beginning of infusion of study medication until the beginning of a persistent beneficial effect, based on the patient's responses to a Treatment Effect Questionnaire for the primary attack location. RUCONEST was generally well tolerated in this Phase III clinical study and the frequency of patients experiencing at least one treatment emergent adverse event in the RUCONEST treated group was less than in the placebo group. Within 72 hours of the completion of infusion of study medication, four RUCONEST patients (7%) experienced six adverse events: sneezing, procedural headache, back pain, skin burning sensation, an increase in fibrin D-dimer and lipoma. Within the 72 hour period four placebo patients (22%) experienced four adverse events: sinus congestion, vasomotor rhinitis, diarrhea and dyspepsia.
surf1944
12 years ago
2:43PM Santarus confirms appellate court reversal in part of ZEGERID invalidity decision (halted) (SNTS) 6.39 +0.21 : Co confirmed that the U.S. Court of Appeals for the Federal Circuit reversed in part a lower court decision of invalidity involving certain asserted patent claims covering ZEGERID(R) Capsules and ZEGERID Powder for Oral Suspension. The Federal Circuit found that certain claims of asserted U.S. Patent numbers 6,780,882 and 7,399,772, which Par Pharmaceutical (PRX). had been found to infringe, were not invalid due to obviousness. The Federal Circuit also upheld the District Court's finding that there was no inequitable conduct. The case will be remanded back to the U.S. District Court for the District of Delaware for further proceedings consistent with the appellate decision. We plan to aggressively pursue all remedies available to us, including damages as well as seeking an order halting further sales of Par's generic product."
surf1944
12 years ago
Santarus to Hold First Quarter 2012 Financial Results Conference Call on May 8
Business WirePress Release: Santarus, Inc. – Thu, Apr 26, 2012 4:00 AM EDT
SAN DIEGO--(BUSINESS WIRE)--
Santarus, Inc. (SNTS - News) today announced that it will release first quarter 2012 financial results after market close on Tuesday, May 8, 2012. Santarus management will host an investment-community conference call at 4:30 p.m. Eastern time (30 p.m. Pacific time) to discuss the financial results and provide a corporate update.
Individuals interested in participating in the conference call may do so by dialing 888-803-8275 for domestic callers, or 706-643-7736 for international callers. Those interested in listening to the conference call live via the Internet may do so by visiting the Investor Relations section of the company’s website at www.santarus.com.
A webcast replay will be available on the Santarus website for 14 days. A telephone replay will be available for 48 hours following the conclusion of the call by dialing 855-859-2056 for domestic callers, or 404-537-3406 for international callers, and entering reservation code: 70617723.
About Santarus
Santarus, Inc. is a specialty biopharmaceutical company focused on acquiring, developing and commercializing proprietary products that address the needs of patients treated by physician specialists. The company’s current commercial efforts are focused on GLUMETZA® (metformin hydrochloride extended release tablets) and CYCLOSET® (bromocriptine mesylate) tablets, which are indicated as adjuncts to diet and exercise to improve glycemic control in adults with type 2 diabetes, and on FENOGLIDE® (fenofibrate) tablets, which is indicated as an adjunct to diet to reduce high cholesterol.
Santarus has a diverse product development pipeline. A New Drug Application for UCERIS™ (budesonide) tablets for induction of remission of mild to moderate active ulcerative colitis is under review by the FDA with a response expected in October 2012. The pipeline also includes two late-stage investigational drugs: RHUCIN® (recombinant human C1 inhibitor) for treatment of acute attacks of hereditary angioedema and rifamycin SV MMX® for treatment of travelers’ diarrhea, both of which are being evaluated in Phase III clinical studies. In addition, the company’s investigational monoclonal antibody, SAN-300, is being evaluated in a Phase I clinical program. More information about Santarus is available at www.santarus.com.
Santarus cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation by Santarus that any of its plans or objectives will be achieved. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in Santarus’ businesses, including, without limitation: difficulties or delays in development, testing, manufacturing and marketing of, and obtaining and maintaining regulatory approvals for, Santarus’ products; and other risks detailed in prior press releases as well as in public periodic filings with the Securities and Exchange Commission.
You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Santarus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
Santarus®, FENOGLIDE® and UCERIS™ are trademarks of Santarus, Inc. GLUMETZA® is a trademark of Biovail Laboratories International S.r.l. licensed exclusively in the United States to Depomed, Inc. CYCLOSET® is a trademark of VeroScience LLC. MMX® is a trademark of Cosmo Technologies Limited. RHUCIN® is a trademark of Pharming Group NV.
Contact:
Santarus, Inc.
Martha L. Hough, 858-314-5824
VP Finance & Investor Relations
or
Debra P. Crawford, 858-314-5708
Chief Financial Officer
or
Investor Contact:
LHA
310-691-7100
Jody Cain, jcain@lhai.com
Bruce Voss, bvoss@lhai.com
@LHA_IR_PR
biginvestors
13 years ago
November 4, 2011
Santarus and Pharming Announce Presentation of Retrospective Analyses of Data from Clinical Studies of RHUCIN
Findings to be presented at the annual meeting of the American College of Allergy, Asthma & Immunology
SAN DIEGO & LEIDEN, Netherlands--(BUSINESS WIRE)-- Specialty biopharmaceutical company Santarus, Inc. (NASDAQ: SNTS) and biotech company Pharming Group NV (NYSE Euronext: PHARM) today announced that a retrospective analysis of integrated efficacy data from patients with Hereditary Angioedema (HAE) undergoing treatment with the investigational drug RHUCIN® (recombinant human C1 inhibitor, or C1INH) for acute attacks of HAE will be presented in an oral presentation at the 2011 Annual Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in Boston, MA on November 6, 2011. The findings of a separate retrospective analysis of immuno-safety data will be presented in the poster sessions at the ACAAI meeting. The data analyzed were derived from placebo-controlled and open-label clinical studies conducted with RHUCIN in patients with HAE.
"We were pleased to have the integrated efficacy data accepted for oral presentation and to have the opportunity to present the poster on immuno-safety of RHUCIN at this major U.S. medical meeting of physicians who treat patients with HAE," said Rienk Pijpstra, MD, MBA, Chief Medical Officer at Pharming.
The abstract Clinical Efficacy of Recombinant Human C1 Inhibitor in North American Patients with Acute Hereditary Angioedema Attacks (R. Levy, et al) is scheduled for an oral presentation at the ACAAI meeting. This integrated efficacy dataset included 70 HAE patients treated for 179 angioedema attacks with 50 U/kg RHUCIN, including patients treated for repeated attacks. Median time to the primary endpoint of onset of symptom relief was 60 minutes and median time to minimal symptoms was 240 minutes. Abdominal attacks had the fastest onset of relief (36 min), followed by urogenital (56 min), oro-facial-pharyngeal-laryngeal (65 min), and peripheral attacks (84 min). In addition, 96% of attacks (172/179) had a clinical response within 4 hours.
A second abstract Immuno-safety of Recombinant Human C1 Inhibitor in Patients with Hereditary Angioedema: An Integrated Analysis (C. Hack, et al) will be discussed in a poster presentation. Data from 155 patients in the RHUCIN acute treatment studies with 424 administrations of RHUCIN were analyzed. The frequency of anti-C1INH antibodies was low and similar in pre- and post-exposure samples (1.7% and 1.8%, respectively). Occurrence of anti-C1INH antibodies did not correlate with repeated treatment or time since last treatment. No neutralizing antibodies were detected. A total of 5/155 (3%) RHUCIN-treated patients had confirmed anti-host-related impurities (HRI) antibodies, which included one patient with anti-HRI antibodies prior to exposure to RHUCIN. The presence of anti-C1INH and anti-HRI antibodies was not associated with clinical symptoms.
RHUCIN Phase III Study
Pharming is conducting a Phase III clinical study with RHUCIN under a Special Protocol Assessment (SPA) that is intended to support the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). RHUCIN is being evaluated for the treatment of acute attacks of angioedema in patients with HAE in an international, multicenter, randomized, placebo-controlled Phase III study at a dosage strength of 50 U/kg with a primary endpoint of time to beginning of relief of symptoms. Santarus has licensed certain exclusive rights from Pharming to commercialize RHUCIN in North America for the treatment of acute attacks of HAE and other future indications. Under the terms of the license agreement, a $10 million milestone is payable to Pharming upon successful achievement of the primary endpoint of the Phase III clinical study. The study is expected to be completed by the third quarter of 2012.
About RHUCIN (RUCONEST® in European countries) and Hereditary Angioedema
RHUCIN (INN conestat alfa) is a recombinant version of the human protein C1 inhibitor (C1INH). RHUCIN is produced through Pharming's proprietary technology in milk of transgenic rabbits and in Europe is approved under the name RUCONEST for treatment of acute angioedema attacks in patients with HAE. RHUCIN has been granted orphan drug designation in the U.S. for the treatment of acute attacks of HAE, a genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in unpredictable and debilitating episodes of intense swelling of the extremities, face, trunk, genitals, abdomen and upper airway. The frequency and severity of HAE attacks vary and are most serious when they involve laryngeal edema, which can close the upper airway and cause death by asphyxiation. According to the U.S. Hereditary Angioedema Association, epidemiological estimates for HAE range from one in 10,000 to one in 50,000 individuals.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of unmet medical needs. RUCONEST® (RHUCIN® in non-European territories) is a recombinant human C1 inhibitor approved for the treatment of angioedema attacks in patients with HAE in all 27 EU countries plus Norway, Iceland and Liechtenstein, and is distributed in the EU by Swedish Orphan Biovitrum. The product is also under development for follow-on indications, i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF) following kidney transplantation. The advanced technologies of the Company include innovative platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products. Additional information is available on the Pharming website, www.pharming.com.
About Santarus
Santarus, Inc. is a specialty biopharmaceutical company focused on acquiring, developing and commercializing proprietary products that address the needs of patients treated by physician specialists. The company's current commercial efforts are focused on GLUMETZA® (metformin hydrochloride extended release tablets) and CYCLOSET® (bromocriptine mesylate) tablets, which are indicated as adjuncts to diet and exercise to improve glycemic control in adults with type 2 diabetes.
Santarus also has a diverse development pipeline, including three investigational drugs in Phase III clinical programs: UCERIS™ (budesonide) Tablets for induction of remission of active ulcerative colitis, RHUCIN® (recombinant human C1 inhibitor) for treatment of acute attacks of hereditary angioedema and rifamycin SV MMX® for treatment of travelers' diarrhea, in addition to other earlier-stage development programs. More information about Santarus is available at www.santarus.com
surf1944
13 years ago
Santarus and Pharming Announce Special Protocol Assessment (SPA) Agreement with FDA for RHUCIN Phase III Clinical Study
Press Release Source: Santarus, Inc. On Thursday August 4, 2011, 1:00 am
SAN DIEGO & LEIDEN, Netherlands--(BUSINESS WIRE)-- Specialty biopharmaceutical company Santarus, Inc. (NASDAQ:SNTS - News) and biotech company Pharming Group NV (NYSE Euronext: PHARM) today announced that they have reached agreement with the U.S. Food and Drug Administration (FDA) on the design of a Phase III clinical study with the investigational drug RHUCIN® (recombinant human C1 inhibitor) under the Special Protocol Assessment (SPA) process that is intended to support the submission of a Biologics License Application (BLA).
RHUCIN is being evaluated for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) in an international, multicenter, randomized, placebo-controlled Phase III study at a dosage strength of 50 U/kg with a primary endpoint of time to beginning of relief of symptoms. The Phase III study was initiated in February 2011, but the FDA subsequently requested modifications to the study protocol.
Following discussions with the FDA and implementation of the Agency’s recommended changes to the study protocol, the FDA has confirmed that Pharming’s proposed trial design, clinical endpoints and statistical analyses are acceptable to the FDA. As a result of the discussions with the FDA, the changes to the study design include, as previously announced, a modification to the way the primary endpoint will be assessed and an increase in the number of patients from 50 to approximately 75. The protocol will also be changed to allow the introduction of open-label doses of RHUCIN as a rescue medication. The study is still expected to be completed by the third quarter of 2012.
“We are pleased to have reached agreement with the FDA under an SPA on the protocol for the Phase III clinical study to support a BLA for RHUCIN in the U.S. Over the past months we have continued to open additional investigational sites and to screen patients for eligibility who can now be randomized into the amended trial,” said Rienk Pijpstra, MD, MBA, Chief Medical Officer at Pharming.
Santarus has licensed certain exclusive rights from Pharming to commercialize RHUCIN in North America for the treatment of acute attacks of HAE and other future indications. Under the terms of the license agreement, a $10 million milestone is payable to Pharming upon successful achievement of the primary endpoint of the Phase III clinical study.
About the Special Protocol Assessment Process
The Special Protocol Assessment (SPA) process is a procedure by which the FDA provides official evaluation and written guidance on the design of proposed protocols that are intended to form the basis for a BLA or New Drug Application (NDA). Final marketing approval depends on the results of efficacy, the adverse event profile and an evaluation of the benefit/risk of treatment demonstrated in all the data contained in the BLA or NDA submission.
About Rhucin (Ruconest in European countries) and Hereditary Angioedema
RHUCIN (INN conestat alfa) is a recombinant version of the human protein C1 inhibitor (C1INH). RHUCIN is produced through Pharming’s proprietary technology in milk of transgenic rabbits and in Europe is approved under the name RUCONEST® for treatment of acute angioedema attacks in patients with HAE. RHUCIN has been granted orphan drug designation in the U.S. for the treatment of acute attacks of HAE, a genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in unpredictable and debilitating episodes of intense swelling of the extremities, face, trunk, genitals, abdomen and upper airway. The frequency and severity of HAE attacks vary and are most serious when they involve laryngeal edema, which can close the upper airway and cause death by asphyxiation. According to the U.S. Hereditary Angioedema Association, epidemiological estimates for HAE range from one in 10,000 to one in 50,000 individuals.