Clinical trial transparency "steadily" improving

by Amy Schofield 24. November 2016 12:51

Pharmaceutical companies are showing a ‘significant trend’ towards increased clinical trial transparency, according to a new ABPI study.

The study shows results of 90% of clinical trials on all 34 new medicines approved by the European Medicines Agency (EMA) in 2013 were disclosed within a 12-month timeframe.

'Clinical trial transparency of recently approved medicines', is published in the peer-review journal Current Medical Research and Opinion (CMRO), and is the latest in a series spanning a five-year period.

Together with results from two earlier studies, this research, conducted by Livewire Editorial Communications on behalf of the ABPI, indicates that the disclosure rate of industry-sponsored clinical trials at 12 months has steadily improved year on year from 71% in 2009 to 90% in 2013.

This study assessed all completed company-sponsored trials related to new medicines approved by the EMA in 2013 and shows that of the 539 evaluable company-sponsored clinical trials related to 34 new medicines licenced to 24 different companies:


  • 484 (90%) had been disclosed on a registry or in scientific literature within 12 months of first regulatory approval or trial completion.
  • 500 (93%) had results disclosed by the end of the study at 31 July 2015.
  • Larger, phase III trials reached disclosure rates of 93% (210/225) within 12 months and 95% (214/225) at 31 July 2015.
Dr Jacintha Sivarajah, Head of Medical Affairs at the ABPI, said: “These latest findings highlight the trend towards increasing rates of disclosure for pharmaceutical industry-sponsored clinical trials, reaffirming the industry’s commitment to greater transparency across all of its relationships and activities."

72% (28/39) of the undisclosed trials were early phase I or II trials. Many of these were initiated more than 10 years ago, prior to the publication of industry commitments, implementation of regulatory requirements and the availability of clinical trial registries, and at a time when they would not have been expected to be published as individual studies.


Dr Bryan Deane, co-author of the report, said: “As long as medicines that began development before 2005 are still reaching approval the disclosure rate is unlikely to reach 100%. These early stage trials pre-date industry’s commitment to greater transparency. 

“However, now that registration and reporting of clinical trials has become routine and industry, particularly large multinational companies, is increasingly focussing on ensuring its transparency requirements are fulfilled, it is reasonable to expect that the trend for increased disclosure rates will continue.”


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PharmaMar seeks European marketing authorisation for multiple myeloma treatment

by Amy Schofield 23. September 2016 08:32

PharmaMar has announced the submission to the European Medicines Agency of the marketing authorisation application (MMA) for Aplidin® (plitidepsin) in combination with dexamethasone for the treatment of relapsed/refractory multiple myeloma.

The application follows positive data obtained from the randomised, Phase III ADMYRE clinical trial, where the efficacy and safety of Aplidin® with dexamethasone versus dexamethasone alone in patients with relapsed/refractory multiple myeloma after at least three, but no more than six, prior therapeutic regimens was evaluated. The results of the ADMYRE study showed a statistically significant 35% reduction in the risk of progression or death over the comparator. The study met its primary endpoint.

Multiple myeloma is a type of blood cancer which represents 10% of all haematological malignancies.

Aplidin® has so far received orphan drug designation by the European Commission and the US Food and Drug Administration.  

PharmaMar expects an answer from the regulatory agency for the second half of 2017.   

AstraZeneca withdraws EU application for experimental cancer drug approval

by Amy Schofield 22. September 2016 11:34

AstraZeneca has reportedly withdrawn an application for European approval to market its experimental cancer drug, cediranib, in combination with chemotherapy, to treat ovarian cancer.

The application is reported to have been pulled because of questions raised at a late stage by regulatory authorities reviewing the product.

The British pharmaceutical company originally sought approval from the European Medicines Agency in June 2015 to sell the treatment for use with platinum-based chemotherapy in adults with a type of ovarian cancer that’s relapsed.

AstraZeneca said on Wednesday that their VEGFR inhibitor cediranib remained an important pipeline medicine and that the decision did not affect its ongoing development in tests in combination with its approved ovarian-cancer treatment Lynparza and the experimental durvalumab cancer therapy. 

A company spokesperson said: “Following lengthy engagement with the EMA, it was clear that there would remain a difference of opinion on the overall benefit-risk of cediranib and on some of the study methods. On balance, we decided to withdraw the application for combination with chemotherapy to focus our attention on combination studies of cediranib with other medicines.”

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CHMP gives nod to Lilly's olaratumab in combination for advanced soft tissue sarcoma

by Amy Schofield 20. September 2016 15:28

Eli Lilly and Company has announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the granting of a conditional marketing authorisation for olaratumab, in combination with doxorubicin, for the treatment of adults in the European Union (EU) with advanced soft tissue sarcoma (STS) not amenable to curative treatment with radiotherapy or surgery and who have not been previously treated with doxorubicin.

If approved, olaratumab will be marketed under the trade name LARTRUVO™. 

This is the first regulatory step in the world towards approval for olaratumab. The CHMP reviewed olaratumab under EMA's accelerated assessment programme and the positive opinion is now referred for final action to the European Commission, which grants marketing authorisation in the EU.

An EC decision on marketing authorisation is expected within two to three months of the CHMP issuing its recommendation.

This will be Lilly's first conditional approval in the EU. As part of a conditional marketing authorisation, Lilly will need to provide results from an ongoing Phase 3 study, ANNOUNCE, which is fully enrolled. Until availability of the full data is made, the CHMP will review the benefits and risks of olaratumab annually to determine whether the conditional marketing authorisation can be maintained.

Dr Richard Gaynor, senior vice president of product development and medical affairs for Lilly Oncology, said: "Advanced soft tissue sarcoma is a rare disease that is difficult to treat, and this milestone brings us one step closer to providing physicians in Europe with a new option that they can offer to their patients."

The EU submission is based on data from Phase 2 JGDG, an open-label, randomised trial that compared olaratumab, in combination with doxorubicin chemotherapy, to doxorubicin alone in patients with advanced STS not amenable to curative treatment with surgery or radiotherapy and who have not been previously treated with doxorubicin. Efficacy endpoints included progression-free survival, overall survival and objective response rate. 

The EMA previously granted olaratumab with Orphan Drug Designation for the treatment of soft tissue sarcoma in the EU.

Sanofi and Regeneron's rheumatoid arthritis drug accepted for EU regulatory review

by Amy Schofield 10. August 2016 08:15

Sanofi and Regeneron's new rheumatoid arthritis therapy, sarilumab, has been accepted for European regulatory review.

The European Medicines Agency has accepted the companies' marketing authorisation application for the investigational human monoclonal antibody.

Sarilumab works by targeting the IL-6 receptor and has been developed for the treatment of adult patients with moderately to severely active rheumatoid arthritis.

The therapy has so far successfully been trialled in seven phase III studies, involving in excess of 3300 adult patients, most of whom had failed to respond to previous treatment regimens.

Sanofi Genzyme, the specialty care global business of Sanofi, will commercialise sarilumab if it is authorised. 

A biologics licence application for sarilumab was also accepted for review by the US Food and Drug Administration during the first quarter of 2016. A decision is expected in the next few months. 

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EMA and FDA accept Roche's MS therapy for review

by Amy Schofield 29. June 2016 15:36

The European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) have accepted Roche's OCREVUS® (ocrelizumab) for review.

The EMA has validated Roche's Marketing Authorisation Application (MAA) of OCREVUS® (ocrelizumab) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS) in the European Union (EU). Validation confirms that the submission is complete and signifies the MAA is under review by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The U.S. Food and Drug Administration (FDA) has also accepted for review Roche’s Biologics License Application (BLA) for OCREVUS for the treatment of RMS and PPMS, and has granted the application Priority Review Designation with a targeted action date of 28 December 2016.

If approved by the EMA and FDA for both indications, OCREVUS would be the first and only treatment for both forms of multiple sclerosis (MS), which affect approximately 95% of people at diagnosis.

OCREVUS is the first investigational medicine to significantly reduce disability progression in people with relapsing and primary progressive forms of MS.

Dr Sandra Horning, chief medical officer and head of Global Product Development, said: “We will continue to work closely with the EMA and FDA to bring this investigational medicine to people with MS as quickly as possible.”

The OCREVUS marketing applications are based on positive results from three Phase III studies, which met primary and key secondary endpoints.

AOP Orphan Pharmaceuticals announces progress of pivotal phase III trial

by Admin 5. March 2015 17:18

AOP Orphan has announced completion of recruitment of its pivotal phase III trial PROUD-PV ( to support global licensure of Ropeginterferon alfa 2b.

Ropeginterferon alfa 2b is a novel, long-acting, mono-pegylated Interferon, administered once every 14 days, after achieving therapy response only monthly.

Based on previous phase I/II trials, Ropeginterferon alfa 2b is expected to be safe and effective in the majority of PV patients.

Haematological and molecular responses occurred in most patients, while several patients achieved undetectable mutated JAK2 levels and a complete normalisation of their chromosomal make-up.

Ultimately, this is expected to result in improved tolerability, convenience and compliance and, as a consequence, better long-term treatment outcomes.

Design and endpoints of this trial have been discussed and agreed with both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA), to support global licensure of Ropeginterferon alfa 2b, which also has Orphan Drug status in both Europe and the USA.

AOP Orphan has exclusively licensed Ropeginterferon alfa 2b for development and commercialisation in the field of Myeloproliferative Neoplasms (MPNs) from Pharmaessentia Corp. a biotech company based in Taiwan for European, CIS, and Middle Eastern markets. Pharmaessentia retains the rights for development and commercialization of P1101 in other major markets, such as North America, Asia, and South America.

Since its commencement in October 2013 over 260 PV patients have been recruited in 50 centres across Europe. Enrolment of patients was successfully completed in February 2015.

Treatment with Ropeginterferon alfa 2b is expected to be safe and effective in the majority of patients and to be superior to hydroxyurea.

Professors Jean-Jacques Kiladjian from Paris and Heinz Gisslinger from Vienna said: “We already know from several smaller studies that interferons work effectively against myeloproliferative diseases.”

Dr. Rudolf Widmann, CEO of AOP Orphan, said: “The unparalleled quick uptake and progress of PROUD-PV, completing enrolment of over 260 patients in around 15 months, proves the eagerness of both patients and physicians for this new treatment paradigm.”

Janssen receives positive CHMP opinion for VELCADE® (bortezomib)

by Admin 22. December 2014 15:47

Janssen has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of VELCADE® (bortezomib) for use in Mantle Cell Lymphoma.

The drug has been recommended in combination with rituximab, cyclophosphamide, doxorubicin and prednisone, for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are unsuitable for haematopoietic stem cell transplantation.

The positive opinion of the CHMP was based on data from the Phase 3 study, LYM-3002. In the European Union (EU), VELCADE is currently indicated for the treatment of multiple myeloma (MM), another rare blood cancer, either as monotherapy or in combination with of other treatment regimens.

MCL is a rare and aggressive type of blood cancer that can be challenging to treat and is associated with a poor prognosis.

Thomas Stark, Vice President, Medical Affairs, Janssen Europe, Middle East and Africa (EMEA), said: "At Janssen, we are committed to continuously developing therapeutic solutions to treat relevant, haematologic diseases like MCL.

"This positive opinion brings us one step closer to offering additional treatment options with VELCADE for patients and physicians, and we are delighted with this recommendation."

The CHMP's positive opinion will now be reviewed by the European Commission, which has the authority to grant a label extension for medicines in the European Economic Area. A final decision on VELCADE's use in MCL by the European Commission is anticipated early next year.

Bayer HealthCare develops new treatment for haemophilia A

by Admin 5. December 2014 16:17

Bayer HealthCare has developed a promising new recombinant factor VIII product for the treatment of haemophilia A in adults and children but is waiting European approval.

The application for marketing authorisation has been submitted to the European Medicines Agency for BAY 81-8973 based on positive results from three phase III clinical trials, involving a total of 2014 subjects.

Clinical Studies into the drug show high-levels of efficacy when used for prophylaxis twice or three times per week with standard dosages. Its capacity for flexible dosing gives greater choice in finding the right option for individual patients.

Bayer also plans to file for approval of BAY 81-8973 in other countries in the coming months.

Dr Joerg Moeller, member of the Bayer HealthCare executive committee and head of global development, said: "The submission of BAY 81-8973 is an important step forward in our efforts to improve treatment options for people with haemophilia."

The company started work on expanding its manufacturing capabilities for haemophilia A products in Germany earlier this year in order to support the production of this therapy, as well as the long-acting agent BAY 94-9027.

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Novartis' new heart failure drug to get accelerated EU review

by Admin 1. December 2014 09:51

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has granted accelerated assessment status to Novartis’ investigational heart failure drug, LCZ696.

The new heart failure drug, intended for patients with heart failure with reduced ejection fraction, is to benefit from an accelerated European review process which will allow the CHMP to grant an opinion after 150 days instead of the normal 210.

Accelerated review status is seldom granted and has never before been awarded to a cardiovascular therapy. A decision on EU approval is now expected to arrive during 2015. 

The regulatory submission will be based on results from the largest ever landmark study conducted in heart failure, PARADIGM-HF. The results showed that LCZ696 can significantly reduce the risk of cardiovascular death or heart failure hospitalisation compared to an established alternative.

David Epstein, division head of Novartis Pharmaceuticals, said: "Novartis is committed to extending and improving more lives sooner with LCZ696, and this decision by the CHMP we hope will greatly support our effort to do so in Europe."



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