Month: November 2013

Mexico at the vanguard with new combination drug

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Regulatory Affairs in Latin America

In the past days, COFEPRIS granted approval for the combination drug containing fluticasone furoate and vilanterol, indicated for treatment of chronic obstructive pulmonary disease (COPD) and asthma. Although the product had already been approved in the US by the FDA in May 2013, for the treatment of COPD, Mexico was the first country worldwide to approve the drug for COPD + asthma simultaneously.

This was a possibility regulatorily, and practically times were so short, (the process took 18 months less than what it would have normally taken) thanks to the Agreement for Innovation, effort of the regulatory authority to promote access of new innovative drugs to the local Mexican market.

Just a couple of days after the product was approved in Mexico, the European Commission, following evaluation by the European Medicines Agency (EMA), granted marketing authorization for the drug for both indications in 31 European countries.

The drug product, developed by…

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Argentina: Site Master File now required by ANMAT for national sites

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Regulatory Affairs in Latin America

Starting on November 19th, 2o13, sites in Argentina approved by ANMAT to manufacture or package, in own or contract facility, or import or export, both drug products and APIs, have to submit their Site Master Files (SMF) annually. This is stated in the new disposition 7066/13 that you can access here (in Spanish).

The file has to be submitted to the front desk of the National Institute of Medicines (INAME) in the format of a PDF file in a CD or DVD. Paper submissions are not required nor accepted. All critical changes to the SMF are to be notified with a new SMF.

Companies have 30 days from publication of the regulation to comply and present their SMF. For the annual submission, new SMFs have to be sent 12 months from the last submission.

An annex specifying the required content for the SMF is attached to the new regulation. Here…

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Orphan Drug Designation Granted for Epidiolex in Dravet syndrome by the FDA

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New Drug Approvals


Cannabidiol

Seven Expanded Access INDs granted by FDA to U.S. 
physicians to treat with Epidiolex 125 children suffering 
from intractable epilepsy syndromes -

LONDON, Nov. 15, 2013

GW Pharmaceuticals plc (AIM: GWP, Nasdaq: GWPH, “GW”) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Epidiolex(R), our product candidate that contains plant-derived Cannabidiol (CBD) as its active ingredient, for use in treating children with Dravet syndrome, a rare and severe form of infantile-onset, genetic, drug-resistant epilepsy syndrome. Epidiolex is an oral liquid formulation of a highly purified extract of CBD, a non-psychoactive molecule from the cannabis plant. Following receipt of this orphan designation, GW anticipates holding a pre-IND meeting with the FDA in the near future to discuss a development plan for Epidiolex in Dravet syndrome.

Dravet syndrome is a rare pediatric epilepsy syndrome with a distinctive but complex electroclinical presentation. Onset of Dravet…

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Mast Ischemia Drug Gets Orphan Drug Designation

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New Drug Approvals

Wed, 11/13/2013
Mast Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has designated MST-188 for the treatment of acute limb ischemia as an orphan drug.http://www.dddmag.com/news/2013/11/mast-ischemia-drug-gets-orphan-drug-designation

MST-188 (purified poloxamer 188)

MST-188 is a purified form of a nonionic, triblock copolymer (poloxamer 188). It is an investigational agent that binds to hydrophobic surfaces on damaged cells and improves membrane hydration and lowers adhesion and viscosity, particularly under low shear conditions. MST-188 has the potential to reduce ischemic tissue injury and end-organ damage by restoring microvascular function, which is compromised in a wide range of serious and life-threatening diseases and conditions. We initially are developing MST-188 as a treatment for complications arising from sickle cell disease.

How MST-188 Works…

Background

Non-purified forms of poloxamer 188 (P188) have been used in foods, drugs and cosmetics since the 1950s. In the 1980s, extensive research on the mechanisms and potential clinical…

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QbD: new Guidance from EMA and FDA

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QbD: new Guidance from EMA and FDA

The European Medicine Agency (EMA) and the U.S. Food and Drug Agency (FDA) have published a joint question-and-answer document that provides further guidance on the quality-by-design concept. Read more.

http://www.gmp-compliance.org/ecanl_654_0_news_4015_8280,8356,Z-PDM_n.html

The Quality by Design (QbD) concept has been introduced by the International Conference on Harmonisation (ICH) document  Q8, supported by Q9, Q10 and also Q11. In the meantime it is well-known within the pharmaceutical industry. However many companies still struggle with the implementation. Both the European Medicine Agency (EMA) and the U.S. Food and Drug Agency (FDA) are supporting the pharmaceutical industry in the employment of the paradigm. Now, EMA and FDA have published a second joint question-and-answer document that provides further guidance on the quality-by-design concept.

The new document focuses on ‘design space verification’ and reflects conclusions reached in the on-going parallel assessment, which was launched in 2011. The objective of the parallel assessment is to share knowledge, facilitate a consistent implementation of the international guidelines on the implementation of the quality-by-design concept and promote the availability of medicines of consistent quality throughout the European Union (EU) and the USA.

The EMA and the US FDA will publish further conclusions on other quality-by-design-related topics as the pilot programme continues and more parallel assessments are conducted.

Source: EMA press release

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2013/11/news_detail_001941.jsp&mid=WC0b01ac058004d5c1

04/11/2013

European Medicines Agency and US Food and Drug Administration release further guidance on quality-by-design approach

The European Medicines Agency (EMA) and the United States Food and Drug AdministrationExternal link icon (US FDA) have published a second joint question-and-answer document that provides guidance on the quality-by-design concept.

Quality-by-design is a science and risk-based approach to pharmaceutical development and manufacturing. It involves designing and developing pharmaceutical formulations and manufacturing processes to help ensure product quality; these concepts are described in the international guidelines ICH Q8, Q9, Q10 and Q11.

The document published today focuses on ‘design space verification’, a demonstration that the combination of the process parameters and material attributes established at pilot scale during pharmaceutical development are capable of delivering a product of appropriate quality on a commercial scale.

In March 2011, the EMA and US FDA launched a three-year pilot programme for the parallel assessment of certain quality or chemistry manufacturing and control (CMC) sections of applications that are relevant to quality-by-design. With the agreement of the applicants, experts from the Japanese Pharmaceuticals and Medical Devices AgencyExternal link icon (PMDA) participate as observers in the programme.

The objective of the parallel assessment is to share knowledge, facilitate a consistent implementation of the international guidelines on the implementation of the quality-by-design concept and promote the availability of medicines of consistent quality throughout the European Union (EU) and the USA.

The question-and-answer document published today reflects the conclusions reached by the EU and US regulators on the specific topic of design space verification as part of their parallel assessment.

The EMA and the US FDA will publish further conclusions on other quality-by-design-related topics as the pilot programme continues and more parallel assessments are conducted.

The pilot programme is open to selected procedures, including applications for initial marketing authorisations, type-II variations and scientific advice. Participation in the pilot is voluntary. Interested applicants and sponsors should notify both agencies three months prior to submission of an application.

Rolapitant , NDA IN 2014

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New Drug Approvals

ROLAPITANT HYDROCHLORIDE

  • Rolapitant HCl
  • Rolapitant hydrochloride
  • Sch 619734
  • SCH619734
  • UNII-57O5S1QSAQ

(5S ,8S)-8-[[(1R)-1-[3 ,5-
Bis(trifluoromethyl)phenyl] ethoxy] methyl]-8-phenyl-1,7-
diazaspiro[4.5]decan-2-one hydrochloride monohydrate. 
  

CAS 914462-92-3

Empirical Formula: C25H26F6N2O2 · HCl · H2O 

Molecular Weight:  555 

USAN Name: Rolapitant hydrochloride 

INN Name:  rolapitantum or rolapitant 

phase 3

CAS Number: 552292-08-7 (rolapitant free base); 914462-92-3 (rolapitant HCl monohydrdate).

It is in late-stage trials of its drug rolapitant, which showed promising mid-stage results in reducing nausea and vomiting in patients undergoing chemotherapy

Rolapitant hydrochloride is a tachykinin neurokinin 1 (NK1) antagonist in phase III clinical trials at Tesaro for the prevention of chemotherapy-induced nausea and vomiting (CINV). Phase II clinical trials are also under way at OPKO for this indication. At Merck & Co., phase II clinical studies were also under way for the treatment of chronic idiopathic cough and for the prevention of chemotherapy-induced nausea; however, no recent developments have been…

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