Since a lot of time will be needed to analyze the data from the trial after its completion, the company decided that there is no need to keep its 510(K) submission pending during this time. As a result, it withdrew its submitted 510(K) application and plans to submit a new 510(K) application only when results from the supplementary trial are available. If approved by the FDA, AppyScore test would be the first test of the blood that can diagnose whether patients are suffering from acute appendicitis.

Previously, AspenBio Pharma has used an analysis of the data received before the supplementary trial and the information from several clinical experts, who have been hired by the company’s regulatory consultants, to draw up the Statistical Analysis Plan (SAP) for the ongoing trial. The plan specifies when the trial is going to end and what objectives it should achieve. However, the SAP provides only an “interim analysis” of the results achieved from the trial so far.

After the news of the withdrawal of the 510(K) submission was released, Daryl J. Faulkner, who is the company’s CEO, said:

“We believe AppyScore has the potential to help improve physicians’ evaluation of appendicitis, eliminating unnecessary CT scans and reducing radiation exposure risk for patients — while also reducing health care costs. We continue to actively enroll patients in the ongoing clinical trial and believe that submitting a new 510(k) based on a full analysis of the data from this study will provide the most effective path to 510(k) clearance. Our new timeline includes finalizing the trial results and submitting the new 510(k) in the second quarter of 2010.”

The Phase 3 trial was named as SUCCEED (Sarcoma Multi-Center Clinical Evaluation of the Efficacy of Ridaforolimus). About 650 patients – who are suffering from metastatic sarcoma but who have previously resorted to chemotherapy for treatment – participated in the trial. The large number of study participants and also the long duration makes the Phase 3 trial the “largest” trial on any drug treating sarcomas.

Right after the news of the completion of participant enrollment was released, Harvey J. Berger, M.D, who is the Chief Executive Officer as well as the Chairman of Ariad, said:

“We are extremely proud of this accomplishment. Achieving full patient enrollment in the SUCCEED clinical trial was a major global undertaking, and our many R&D groups deserve tremendous recognition for their work. We are very pleased to have achieved this milestone in the timeframe that we established at the outset and now look forward to completing the trial and reporting data from the study next year.”

The company aims to achieve the same results as it did in Phase 2a trial. It also plans to have a detailed analysis of the results obtained in Phase 2a trial. Both the trials targeted the same segment of patients – that is, people suffering from lung transplant. Meanwhile, the company has confirmed that all the trials and other development activities for RSV01 have been solely funded by Alnylam itself – and not by its partner Cubist. However, the two partners have agreed to equally share the costs associated with developing ALN-RSV01. One thing to note here is that Kyowa Hakko Kirin Co., Ltd. – which is the company’s partner in the region of Asia – is not providing any help with funding.

The reason why Alnylam alone fund the development of RSV01 can be attributed to its strong financial position. Patricia Allen, who is the Vice President of the company’s Finance and Treasurer Department, said:

“We are on track to meet our guidance of ending 2009 with a cash position of greater than $430 million, a financial position that allows us to invest in the advancement of our platform and pipeline of novel RNAi therapeutic products. We expect to finish 2010 with greater than $325 million in cash, which excludes the potential payment from Novartis should they decide to execute their adoption license later this year.”

The Phase 2 trial took place in the city of Perth, Australia. More than 200 people in healthy conditions participated in the trial. All these participants were given doses of oral interferon once every day. The doses were given for a period of sixteen weeks. Then the patients were observed for another four weeks.

The main objective of the trial was finding whether study participants suffered from less frequent sicknesses associated with the respiratory symptom or not – especially during the season when flu or cold is most prevalent. The trial also tried to find whether the drug alleviated the flu symptoms in a few patients who do catch flu. Manfred Beilharz , who is an Associate Professor in the department of Microbiology and Immunology Discipline (BBCS), the University of Western Australia, played a key role in conducting the trial. The company also acknowledges the fact that the trial could never have been a reality if it was not funded by the Department of Health, Government of Western Australia.

The Cobalamin drug delivery technology makes use of the natural body mechanism through which vitamin B12 taken orally can be absorbed. The product is based on the innovative idea that the same body mechanism can help the absorption of oral drugs. In fact, the drug absorption can become a lot faster – a phenomenon called “Trojan horse”. This drug delivery technology is used for the following types of drugs: insulin, hormone causing growth in humans, and two types of peptides which cannot be taken orally and thus must be injected into the body.

While discussing the current and potential applications of the drug delivery technology, David P. Nowotnik, Ph.D., who is the Senior Vice President of the Research & Development department of Access Pharmaceuticals, said:

“While Access’ focus has been on the oral delivery of peptides, the technology is sufficiently flexible to allow us to deliver a wide range of actives. In addition to peptide delivery, we have received inquiries recently about the potential of this technology to deliver actives ranging from small molecules to siRNA to monoclonal antibodies. As siRNA needs to be delivered intracellularly to be effective as a therapeutic, the Cobalamin technology may be particularly beneficial as an intracellular delivery technology, as the demand for vitamin B12 increases in many disease states.”

The regulator also handed out a Prescription Drug User Fee Act date – also called Action date or just PDUFA date – of December 24, 2009. This was the date on which the FDA was expected to complete the Priority Review and to provide a response on the NDA to Salix. The stakeholders of Salix were eagerly looking forward to this date. Although the date got extended to February 23, 2010, the final results were favorable for Salix. XIFAXAN^® was clearly approved by the FDA’s Gastrointestinal Drugs Advisory Committee. 14 members of the committee voted for XIFAXAN® – compared with only 4 members who did not approve the drug. Salix also disclosed that the regulator had provided it with an Action date of March 24, 2010. This is the date by which the FDA will finish reviewing the complete NDA application and let the company know whether XIFAXAN® can be brought into the US markets or not.

Before providing its decision, the Gastrointestinal Drugs Advisory Committee investigated the results from the Phase 3 trial that Salix had conducted. The trial was “double–blind, placebo–controlled” and measured the safety and the effectiveness of the drug in treating Hepatic encephalopathy (HE). It was conducted in multiple centers in a number of countries throughout the world. About 299 people suffering from HE participated in the trial.

Right after the news of the approval by the committee came out, Bill Forbes, Pharm.D., who is the Chief Development Officer (CDO) and also the Senior Vice President of Salix Pharmaceuticals, said:

“We are very pleased with the advisory committee’s support for the approval of XIFAXAN 550 mg tablets. If approved, XIFAXAN 550 mg will be the first new option for the management of hepatic encephalopathy in over 30 years. We believe the availability of XIFAXAN 550 mg has the potential to change the treatment paradigm for HE. Today’s independent recommendation from the outside experts comprising the advisory committee reinforces the Company’s confidence in the potential for XIFAXAN 550 mg to provide a solution for patients suffering from this serious condition.”

The FDA pivot trials took place in six different clinics of the United States. About 2000 women participated in those trials. According to the company, the trial showed that LightTouch can detect cancerous and pre cancerous cells in the cervix by passing light onto the cervix and then by studying the wavelengths of the reflected light.

In the American Society meeting, the study results will be disclosed though a series of 5 peer-to-peer presentations titled as “Multimodal Spectroscopy as a Triage Test for Women at risk for Cervical Neoplasia: Results of a 1607 Subject Pivotal Trial”, “Multimodal Spectroscopy as a Triage Test for Women at risk for Cervical Neoplasia: Results for Adolescent Subjects”, “Multimodal Spectroscopy as a Triage Test for Women at risk for Cervical Neoplasia: Hispathology Review Procedures and Results”, “Multimodal Spectroscopy as a Triage Test for Women at risk for Cervical Neoplasia: Results of Follow Up Data” and “Multimodal Spectroscopy as a Triage Test for Women at risk for Cervical Neoplasia: Experience with a Low Cost Commercial Prototype”. All these presentations will be done by 5 eminent American doctors.

Apart from the presentations, the company is also planning to display a real LightTouch device. All the components of the device, such as the user interface and the one-time use calibration, will also be presented.

The ODAC is an independent committee whose members or experts are unbiased and not influenced by any pharmaceutical company. When an NDA is submitted to the FDA, the ODAC analyze the information – regarding the safety and the efficiency of the drug – and send its feedback to the FDA. This feedback is one of the tools that the FDA uses to decide whether to approve the NDA or not. However, there have been many cases in which the FDA approved a particular NDA, even if the feedback from the ODAC regarding the application had been negative.

On March 22 – that is, on the ODAC meeting – the experts would check the information related to the efficiency and the safety of the drug from various sources – the applicant or sponsor (that is, ChemGenex), the staff from the FDA who have already conducted a review on the NDA, and other third party experts who specialize in oncology.

Greg Collier, PhD, who is the Managing Director as well as the Chief Executive Officer of ChemGenex, said:

“The ODAC meeting is a significant milestone in the review process for OMAPRO, our team is well-prepared and we are looking forward to presenting to the ODAC panel.”

Meanwhile, the company also sent a variation of the NDA – called the supplemental new drug application (sNDA) to the FDA. This sNDA was submiited to get the regulator’s approval for marketing Nexium® (the brand name for the generic drug esomeprazole magnesium). This drug, as well as the drug containing asipriin/esomeprazole combination, aims to reduce the risks (especially those of duodenal and gastric cancer) associated with using low dose aspirin. The risks are even higher for patients who are being exposed to NSAID (non steroidal anti inflammatory drug) therapy.

Although aspirin can prevent heart diseases, heart attack and brain stroke, it can also cause peptic and stomach ulcers as well as less serious symptoms like stomach pain. Even low-dose aspirin is not safe. Many patients have stopped their NSAID therapy after facing such symptoms. In fact, one out of every three patients is likely to suffer from these symptoms and to discontinue their therapy.

To test the effectiveness as well as the safety of Nexium, the company carried out two trials. One of the trials was carried out on 40 mg and 20 mg of the drug, while the other was carried out on 20 mg of the drug. The 40/20 mg is used to treat erosive esophagitis (EE) for a period of 4 to 8 weeks while the 20 mg Nexium is used to treat EE for a longer period (usually greater than 6 months). Both these trials evaluated Nexium with placebo. The study results are included in the submitted sNDA and NDA

APF530 is made of granisetron, which is a 5-HT3 antagonist. The current FDA regulations allow granisetron oral tablets and injections to be used only in cases of acute onset CINV. However, according to the company, the Phase 3 trial results showed that APF530 could be used for acute onset CINV as well as for delayed onset CINV. The trial was conducted in several centers. A total of 1,395 people suffering from cancer took part in this trial.

Most cancer patients suffer from nausea and vomiting after being exposed to chemotherapy – which is a necessary form of cancer treatment. Acute onset CINV starts the day the patient gets chemotherapy treatment while delayed onset CINV starts much later than that. If the NDA for APF530 gets approved by the FDA, it will be the only drug – other than Palonesetron injections – to be used for delayed onset CINV.

After the news of the FDA’s acceptance for review of the NDA was released, Ronald J. Prentki, who is the CEO and also the President of A.P. Pharma, said:

“The acceptance of the APF530 NDA represents another important step towards providing physicians and patients with a potential new long-acting therapeutic agent to combat chemotherapy-induced nausea and vomiting. Our team recognizes the important role APF530 could play in cancer care, and we are dedicated to working with the FDA as it reviews our NDA submission.”