Monthly Archives: March 2013

March 31, 2013 | By Márcio Barra


As this blog hits its three month birthday, I thought it would be good to explain what is this blog all about, who I am (there’s always the about me link above), the workflow of the blog and some stats.

Pharmupdates curiously started as an idea that I had to share some of my work from the bachelor degree that I had lying around. It was mostly some articles on the number of clinical trials in Portugal, and a complete list of Portuguese clinical trial sponsors from the 2007-2011 periods. These works were never really used the way I had hoped they would be, and so they were left to gather dust in my computer.

During my Master’s classes, I had the pleasure to meet some amazing speakers from the industry, who weren’t shy to share their knowledge and have a good talk in the break. When it was question time, I usually had a question or two for the speaker, who would gladly answer.

Now, in one break, someone asked me: “what do I think of subject x”. This was extremely flattering to me, to think that someone already working, and with years of experience in the industry, would ask me, someone whose only experience comes from books and reading the news, my thoughts on a subject.

Then I started thinking, what if I had a website, or a blog, where I could share some works that I have done, news that I find important on the pharmaceutical industry, or even opinion pieces. Now, opinion pieces are still a ways off, as I don’t think that my current knowledge and experience can sustain a full-fledged opinion piece, save the “I don’t think that Portugal is making a good argument for pharmaceutical companies to invest here”. But I figured I could write about the other two.


And so Pharmupdates was created.


When I started writing for the blog, I noticed that there was a bit of lack of English written news about Portugal. I mean, if you are Portuguese and you want to know the latest local developments of the pharmaceutical industry, it’s easy, you just hit a Portuguese news website and start reading.

But what about foreign readers? If someone from Germany wants to know, for some reason, how are drugs priced in Portugal, what are the new reference countries for Portugal or information on clinical trials, well they are out of luck. So I made a focus to try and write about Portuguese news, instead of just focusing on international news or big pharma news.

As for the results, they are good I would say. This blog has given me a bit of visibility, just a bit though, People come in to this blog not through links, but by searching for a specific tag or subject. WordPress tools allows me to see how do people reach my blog, and it’s mostly through Google search. This leaves me happy, seeing that someone from another country has checked the website, and at bare minimum has read the title. From the specificity of the keywords, it’s safe to assume that they have also read the article, or just the bit they were interested in.

In total, I garnered 1200 views in these three months, with 700 visitors from many countries. For someone who is just starting, only writes a post per day (save a special occasion, like a trip) and usually tries to write it fast, say 30 minutes each day, I’m happy. The time I can spare for the blog may not seem much, but from the other work that I have, this is really what I can. I’m a Master’s student, first year, with a lot of ongoing projects at the moment, while also looking for a curricular internship. Time is a precious resource.

Speaking of curricular interships, say, If you like what you see here in this blog, and my linkedin profile, and wouldn’t mind having someone on your ranks that is very eager to learn and work, send me an e-mail requesting my CV, and I will be happy to send you!

Thanks for stopping by Pharmupdates! I have some original articles on the backburner, and the news section will continue to expand! Check checking for updates! Pharmupdates!

March 29, 2013 | By Márcio Barra

The big news yesterday was that Tecfidera (dimethyl fumarate) from Biogen Idec Ltd, won approval in the US as a first-line oral treatment for people with relapsing forms of MS. Tecfidera was just approved last Friday by EMA, and the US approval is sure to thrill Biogen.

This drug acts primarily by activating the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) transcriptional pathway, which helps protect nerve cells from damage and inflammation, thus easing multiple sclerosis symptoms like muscle weakness and difficulty with coordination and balance.

Biogen has already a strong MS franchise in the US, including the blockbusters Avonex (interferon beta-1a), and Tysabri (natalizumab). Tecfidera is expected to join the ranks of these blockbusters and go face to face with its oral rivals, Novartis’ MS pill Gilenya (fingolimod) and Aubagio, a once-a-day tablet from Sanofi (this one was approved too last week by EMA alongside Tecfidera, although EMA declined to give it a “new active substance” designation, paving the way for generic competition. It is approved in the US since September last year)

hm duno

This drug, due its improved safety profile versus Gilenya and Aubagio, is widely expected to become the No. 1 oral treatment for Multiple Sclerosis, with annual sales topping $3 billion if it gains the trust of MS physicians. Aubagio carries the risk of liver injury, birth defects and comes along with a boxed warning. As to Gilenya, although first to market, it has been held back by heart safety concerns.

Tecfidera only causes a decrease in a person’s white blood cell count, alongside nausea, vomiting, and diarrhea, and these tended to get better over time without stopping the drug.

As for the price, it will be priced at $54,900 per patient per year, cheaper than Gilenya ($60,000 per patient per year), but more expensive than Aubagio ($48,000 per year). Like all MS treatments, patient assistance programs are in place to help pay for the drug when its price is not covered by insurance.



March 28, 2013 | By Márcio Barra

The whole drug price debate in Portugal continues, as the Pharmaceutical Research and Manufacturers of America (PhRMA) association want to place Portugal in a “priority vigilance list”, due to difficulties  American pharmaceutical companies are having in Portugal.

In a report that Lusa News Agency had access, PhRMA states that they, and the companies they represent, are worried with Portugal, as it has “an ineffective patent protection mechanism”, being “very difficult to ensure timely resolution of patent disputes in Portugal”

They are also unhappy with the ongoing cost containment measures and drug expenditure cuts  that are being applied, which “do not make up the value of innovation and deny the benefits that innovative drugs can bring to Portugal”. The report further states that these “budget constraints “are unrealistic, namely the goal of reducing the public expenditure on medicines to 1% of the GDP. These “increasing difficulties” are affecting the launch of many innovative drugs in Portugal.

PhRMA wants to include Portugal in a “priority vigilance list” expecting a “swift and effective” resolution.

João Almeida Lopes, the president of the Portuguese Association of Pharmaceutical Industry (Apifarma), commented to Lusa that the current government measures are “compromising Portugal”, and that he already warned the Portuguese Government to the patent issue.


RCM pharma

March 27, 2013 | By Márcio Barra

Last week there were a bit of bad news for Novartis, as they saw Afinitor being refused by NICE, the UK price regulatory agency.

But in a final guidance published today, NICE has recommended Novartis’ Tobi Podhaler (tobramycin) and Forest Lab’s Colobreathe (colistimethate sodium), two antibiotics, as options for treating certain lung infections (P. aeruginosa) in people with cystic fibrosis. These two drugs are dry powder versions of already available nebulized products.

This recommendation comes with a caveat though, as Novartis will have to provide the drug with a discount, alongside a Patient Access Scheme and only when nebulized Colobreathe is contraindicated, non-tolerated or shows lack of efficacy in a patient. The previous price without a discount for this drug was £1,790 for 56 days of treatment.

Novartis’ Tobi Podhaler was approved for sale in the US last Friday by the FDA, and it’s being touted as the first powder inhaled antibacterial in the US.

Forest Lab’s Colobreathe recommendation follows the same lines, with a discount and a Patient Access Scheme having to be provided. NICE’s recommendation states that Colobreathe should only be used in cystic fibrosis patients who do not tolerate its nebulized form. Colobreathe is currently priced at £1,936 for 56 days of treatment, and received marketing approval by EMA back in February.

Colobreathe Inhaler

Nebulised colistimethate sodium is the first choice for people with cystic fibrosis and infected with P. aeruginosa. The dry powder version of either drug should be considered when lack of efficacy Nebulised colistimethate sodium is deemed inadequate for safety of efficacy reasons. These are surely good news for the 8,000 people in the UK afflicted with Cystic fibrosis, as their treatment options in case of infection increased.



Online Pharmatimes

March 25, 2013 | By Márcio Barra

Last Thursday, Astrazeneca’s new chief executive Pascal Soriot announced its massive re-structuring plan for the company, with 5050 layoffs planned, more focus on the blood thinner Brilinta, and a $240 million bet on a novel but untested technology from a small biotechnology company, Moderna Therapeutics. For this to succeed, revenue has to be secured as the company is struggling with falling sales of other blockbuster medicines that have lost patent protection or will do so soon, posing a major challenge to new CEO Pascal Soriot. And today, good news reached AstraZeneca

AstraZeneca’s CEO Pascal Soriot

Today, via a press release, the company announced that it won a US legal judgment defending the patent of its cholesterol lowering drug Crestor, with generic firms Watson Laboratories, a unit of Actavis, and Egis admitting that the cholesterol drug’s patent is valid. In return, AstraZeneca agreed to share the market starting in May 2, 2016, 67 days before its pediatric exclusivity expires, with the companies having to pay royalties (39% of net sales) to AstraZeneca during that period.

The two generic firms had been trying to circumvent the patent by developing an alternative chemical version of Crestor, rosuvastatin zinc (instead of calcium).

Crestor is a cornerstone of AstraZeneca’s profits, generating $6.2bn for the company last year, although its sales have been hit by patent’s expiries of similar cholesterol lowering treatments like 2011 Lipitor. Stopping the sale of generics of Crestor is a needed move for a company about to undergo deep restructuring

Back in December, AstraZeneca wrapped up patent litigation with Teva Pharmaceutical Industries, following Teva’s patent infringement suit for the rights to Crestor in 2008.

Now, there’s some controversy on whether these deals, dubbed patent settlements, are anticompetitive or monopolizing, alongisde keeping cheaper products off the market. To read more about that, follow this link for an article by Ed Silverman on that topic.



March 25, 2013 | By Márcio Barra

Paulo Macedo, speaking this last friday at the closing session of the symposium celebrating the 25th Anniversary of the Surgery Center cardiothoracic (CCC) of Coimbra’s Hospital and University Center (CHUC), spoke of the benefits the ongoing drug price cuts are bringing to the population.

Paulo Macedo, Portugal’s Health Minister

“If we didn’t have cheaper drugs, we would have people with lots of difficulties” and that the “national interests (…) are not secured by favoring the international pharmaceutical industry, that is for sure”, said the Health Minister

“Having lower price drugs for the population” is one of the ways of securing the nation’s interests stated the minister.

He stressed however that “we cannot go down to certain levels, where the access to innovation is jeopardized.”

This comes as a response to Bial’s CEO Luís Portela declarations that the successive drug price cuts are hurting pharmaceutical companies, including Bial, a national pharmaceutical company, who had to postpone for two year the start of a project for a new drug for Parkinson. While the minister’s comments may indicate that there is a certain threshold where the current drug cost containment policy will stop, Luis Portela’s previous statements back in February indicate that at least the national pharmaceutical industry is being hurt bad, and having troubles pursuing innovation. 



March 22, 2013 | By Márcio Barra

EMA’s CHMP 18 – 21 March meeting was fruitful, with 5 new drugs attaining marketing authorization (MA), 2 MA applications denied, 3 new generics MAs approved, 4 drugs seeing their therapeutic indications extended, and 2 companies giving up on their MA application. Here’s the rundown approvals and refusals:

Approved marketing authorizations for new drugs

Aubagio (teriflunomide), Sanofi-aventis – A selective immunosuppressant, with anti-inflammatory properties for the treatment of multiple sclerosis.

HyQvia (human normal immunoglobulin), Baxter Innovations GmbH – A replacement therapy for adults with primary immunodeficiency syndromes such as:

  • congenital agammaglobulinaemia and hypogammaglobulinaemia
  • common variable immunodeficiency
  • severe combined immunodeficiency
  • IgG subclass deficiencies with recurrent infections

Iclusig (ponatinib), Ariad Pharma Ltd – A protein kinase inhibitor (L01XE24) that acts through inhibition of the BCR-ABL kinase.

Stribild (elvitegravir / cobicistat / emtricitabine / tenofovir disoproxil), Gilead Sciences, Inc. – A combination antiviral for treatment of HIV infections, containing the following active substances:

  • Elvitegravir, an HIV-1  integrase strand transfer inhibitor (INSTI)
  • Cobicistat, which enhances the systemic exposure of elvitegravir
  • Emtricitabine, a nucleoside analogue of cytidine
  • Tenofovir, a nucleoside monophosphate (nucleotide) analogue of adenosine monophosphate.

Tecfidera (dimethyl fumarate), Biogen Idec Ltd – A drug for the nervous system that  primarily acts by activating the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) transcriptional pathway, for the treatment of relapsing remitting multiple sclerosis.

Denied marketing authorizations of new drugs

Defitelio (defibrotide), Gentium SpA – An orphan drug,for the treatment and prevention of hepatic veno-occlusive disease in blood stem cell transplantation therapy. It’s MA was refused on the basis of insufficient evidence of the medicine´s benefit, alongside some doubts on the way the efficacy study was designed.

Labazenit (budesonide / salmeterol), Laboratoires SMB S.A. – A combination drug for asthma, containing Budesonide, a corticosteroid that works as an anti-inflammatory agent, and salmeterol, a long-acting beta-2 agonist. These drugs are commonly found in other products either alone or in combination for the treatment of asthma. It was refused as the study comparing Labazenit with budesonide alone did not show satisfactory efficacy.

Approved marketing authorizations for new generics

Memantine ratiopharm (memantine hydrochloride), Ratiopharm GmbH – A psychoanaleptic antidementia drug, that acts through antagonism of the N-methyl-D-aspartate (NMDA) receptor, controlling the elevated levels of glutamate that may lead to neuronal dysfunction.

Stayveer (bosentan monohydrate), Marklas Nederland BV – An anti-hypertensive drug and endothelin receptor (ETa and ETb) antagonist, which decreases pulmonary vascular resistance. It is used for the treatment of pulmonary arterial hypertension.

Voriconazole Accord (voriconazole), Accord Healthcare Ltd – A broad spectrum, triazole antifungal agent, that works by inhibiting an essential step in fungal ergosterol biosynthesis.  It is for the “Treatment of invasive aspergillosis, treatment of candidemia in nonneutropenic patients, treatment of fluconazole-resistant serious invasive Candida infections (including C. krusei), and treatment of serious fungal infections caused by Scedosporium spp. and Fusarium spp.”

Approved extensions for new therapeutic indications of existing drugs

Mabthera (rituximab), Roche Registration Ltd – This drug is currently used for the treatment of Non-Hodgkin’s lymphoma (NHL), Chronic lymphocytic leukaemia (CLL) and Rheumatoid arthritis. The new approved extension is for granulomatosis with polyangiitis and Microscopic polyangiitis.

Soliris (eculizumab), Alexion Europe SAS – Aka the world’s most expensive drug, priced at more than $409,000 per year, used for the treatment of paroxysymal nocturnal hemoglobinuria in adults only, and atypical haemolytic uremic syndrome (aHUS) in adults and children. The new indication is an extension of the paroxysmal nocturnal haemoglobinuria indication to include children.

Viread (tenofovir disoproxil fumarate), Gilead Sciences International Ltd – For the treatment of Hepatitis B infection. Now it is also indicated for the treatment of chronic hepatitis B in adults with evidence of lamivudine-resistant hepatitis B virus.

Xarelto (rivaroxaban), Bayer Pharma AG – Therapeutic indication for a new 2.5mg dose was approved. It is “for the prevention of atherothrombotic events in adult patients after an acute coronary syndrome (ACS) with elevated cardiac biomarkers”, either “co-administered with acetylsalicylic acid (ASA) alone or with ASA plus clopidogrel  or ticlopidine”

Applications withdrew

Fanaptum (iloperidone), Vanda Pharmaceuticals Ltd – A drug for treatment of schizophrenia in adults. The company withdrew the application because the CHMP identified missing data, which Vanda could not make available within the regulatory timeframe.

OraNera (autologous oral mucosal epithelial cells), CellSeed Europe Ltd – A cell therapy intended for the treatment of limbal stem cell deficiency in adults. The application was withdrawn on the basis of the CAT’s preliminary assessment, as the committee could not agree on a positive benefit-risk balance for the therapy and the company could not bring additional information in an acceptable timeframe.

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