An established blood cancer drug is now available in a subcutaneous form, cutting the administration time from two hours to five minutes.

The subcutaneous (SC) form of Roche’s MabThera (rituximab), used to treat non-Hodgkin lymphoma (NHL), could improve patient experience while freeing up time in chemotherapy suites.

MabThera SC uses a new biological technology from Halozyme that locally and reversibly breaks down sub-skin tissues.

The SABRINA phase III trial compared the effects of MabThera SC and MabThera IV in patients with previously untreated follicular lymphoma.

It proved the non-inferiority of MabThera SC, with an objective response rate (proportion of patients whose cancer shrinks by over 50%) of 90.5% for SC and 84.4% for IV.

Roche has applied to the European Medicines Authority (EMA) for an updated licence to allow NHL patients in the UK to receive MabThera SC.

MabThera IV is the standard treatment for NHL. In MabThera SC the drug is combined with the human enzyme hyaluronidase, which temporarily increases the penetrability of the tissue layer under the skin, allowing rapid absorption.

Dr Andrew Davies, Consultant in Medical Oncology at the University of Southampton, said: “This is a new formulation of a drug we are very familiar with and have been using for many years. In Southampton, we have observed a high degree of patient preference and satisfaction with this new formulation of rituximab.”

NHL is one of the most common cancers in elderly people; over 12,200 people are diagnosed with it each year in the UK.

Halozyme has four technology partnerships involving the use of hyaluronidase to facilitate SC injections. Its partnership with Roche has also produced an SC version of Herceptin.

The company’s other partnerships to exploit this technology are with Baxter Healthcare, ViroPharma and Intrexon.

The first and only C1 inhibitor approved for use in adults and adolescents in the UK with the rare and life threatening genetic disorder, hereditary angioedema (HAE), has been launched.

ViroPharma’s Cinryze (C1 inhibitor [human]) is a new treatment option which has demonstrated in Phase III pivotal trials to significantly reduce the duration and frequency of HAE attacks.

Dr Hilary Longhurst, Consultant Immunologist and Clinical Lead at Bart’s and the London Children’s Hospital, says the availability of Cinryze provides “another important therapeutic option”.

HAE is a rare, debilitating and potentially life-threatening genetic disorder affecting around 10,000 people across Europe. It’s estimated there are around 1,850 cases in the UK.

People living with the disorder suffer from recurrent and disabling attacks of swelling that may affect the larynx, abdomen, face, extremities and urogenital tract.

Cinryze is the first and only C1 inhibitor approved for use in the UK for the prevention of angioedema attacks in patients with severe and reoccurring HAE who are intolerant to or insufficiently protected by oral treatments, or in patients who are inadequately managed with repeat acute treatment.

Eligible patients are able to self-administer the treatment, giving doctors and patients convenience and the ability to better manage the condition.

“We are excited to offer the only C1 inhibitor approved in the UK with a label that includes routine prevention of attacks, in addition to treating the attacks as they occur,” said Matt Mason, ViroPharma’s General Manager, UK and Ireland. “We will be working closely with physicians to help and support patients through prevention and treatment of their HAE attacks.”

The Scottish Medicines Consortium (SMC) has approved Buccolam (midazolam, oromucosal solution) from ViroPharma for emergency treatment of prolonged, acute epileptic seizures in children (aged over three months) and teenagers.

The decision will give youngsters in Scotland an alternative to the standard emergency grand mal treatment, rectally administered diazepam.

Buccolam will be marketed in Scotland by ViroPharma Ltd, a subsidiary of US company ViroPharma Inc.

The SMC considered that Buccolam was at least as effective clinically as rectal diazepam and was more cost-effective.

Buccolam, which has anti-convulsant, sedative and muscle-relaxant effects, can be administered by parents and carers of children with epilepsy.

The medication is administered from a prefilled oral syringe to the space between the cheek and gums, to be absorbed through the lining of the mouth.

While various unlicensed forms of midazolam have been used with children in the UK, Buccolam is the first to have received a Paediatric Use Marketing Authorisation licence (in September 2011).

The licensing of Buccolam follows the advice of MHRA in recent years that “specific children’s-only medicines” should be made available as an alternative to giving children “cut-down doses” of adult medicines.

“The SMC’s approval of Buccolam as an emergency medication is welcome news for hundreds of families and clinicians in Scotland,” commented Lesslie A. Young, Chief Executive of Epilepsy Scotland. “We are delighted that this licensed drug is now available to treat infants to teenagers with severe and prolonged epileptic seizures.”

Thierry Darcis, ViroPharma’s General Manager for Europe, commented: “We are committed to delivering solutions that address critical gaps in care for patients living with few, if any, clinical treatment options.”

Nearly a million children and adolescents in the EU suffer from active epilepsy, which causes life-threatening seizures.

The European Commission (EC) has authorised DuoCort Pharma’s Plenadren (hydrocortisone) to be marketed in the EU for the treatment of adrenal insufficiency.

The dual release hormone replacement therapy is the first pharmaceutical innovation in over 50 years for adults suffering from adrenal insufficiency, or cortisol deficiency.

Professor Gudmundur Johannsson, Chief Medical Officer at DuoCort Pharma, said: “Plenadren offers a welcome new treatment option to help patients suffering from adrenal insufficiency. It can improve therapy for many of the almost 200,000 patients in Europe who suffer from this disease and who need lifelong cortisol replacement therapy for their survival.”

The once-daily tablet is designed to imitate the normal physiological cortisol profile, with an outer layer that releases hydrocortisone immediately into the bloodstream and an inner core releasing the rest of the medicine gradually throughout the day.

Although glucocorticoid replacement therapy for adrenal insufficiency has been available for decades, complications have been linked to the medication, including premature death, impaired quality of life, increased risk of cardiovascular diseases, and decreased bone mineral density.

Adrenal insufficiency (cortisol deficiency) is a rare, life-threatening disease that affects patients in their active years. Patients require lifelong replacement therapy with hydrocortisone, the synthetic form of cortisol, replacing or substituting the hormones that the patient's adrenal glands are not producing.

DuoCort Pharma provides drug development with a focus on improving glucocorticoid therapy. The company is currently being absorbed by ViroPharma, who acquired the pharmaceutical company for an upfront payment of $33 million dollars in October.

ViroPharma has signed a definitive agreement to acquire Swedish-based DuoCort Pharma AB for an initial $33.6 million – but only if Plenadren is approved by the European Commission (EC).

Under the terms of the agreement, the deal will be closed after the tablet’s approval, after the EC’s confirmation of Plenadren’s orphan drug designation, and an amended agreement with the product’s contract manufacturer.

Vincent Milano, ViroPharma's President and CEO, says the deal is in line with the company’s “objective of broadening our orphan drug portfolio”.

Further milestone patients of up to $131 million associated with manufacturing, sales thresholds and territory expansion have also been agreed as part of the acquisition.

Plenadren is a once daily dual-release oral glucocorticoid tablet with a release profile designed to closely mimic the body's natural secretion pattern of cortisol. ViroPharma now anticipates the commercial launch of the treatment – which it says will be the first “true innovation in over 50 years” – of adrenal insufficiency in the EU within a year. It estimates that peak annual sales for Plenadren could reach up to $50 million.

The Committee for Orphan Medicinal Products confirmed in September that Plenadren’s orphan designation would be maintained and protected for a decade of market exclusivity.

ViroPharma’s Buccolam has been specifically licensed for infants, children and adolescents to treat severe convulsions and epileptic seizures in Europe.

It is the first children’s medicine to be granted the Paediatric Use Marketing Authorisation (PUMA), delivering an age-specific dose in the right form for the child.

Dr June Raine, Director of Vigilance and Risk Management of Medicines, MHRA, said: “They are not simply ‘small adults’ and they respond differently to medication”.

The midazolam and oromucosal solution comes in a pre-filled syringe indicated for patients aged three months to 18 years of age.

The dose is administered via the cavity between the cheek and gum, a less invasive method than its current treatment option, which is rectally-administered diazepam.

The marketing authorisation is based on data gathered from four clinical trials testing Buccolam against diazepam, where Buccolam proved to be either comparable to, or better than the existing treatment.

“Children should have access to medicines that have been especially designed for them,” added Dr Raine.

The medicine previously received a positive opinion in June from the Committee for the Medicinal Products for Human Use (CHMP) and will now be available across the EU as well as Norway, Iceland and Liechtenstein.

Thierry Darcis, Vice President and General Manager of ViroPharma Europe, said: “We look forward to working closely with physicians across Europe as we launch Buccolam into the European markets”.

ViroPharma’s Buccolam (midazolam, oromucosal solution) has been given a positive opinion by the CHMP for the treatment of seizures in patients aged three months up to 18 years old.

The treatment, if approved by the European Commission, would become the first product approved through the centralised ‘Pediatric Use Marketing Authorization’ (PUMA) procedure.

Professor Ian Wong, Director of the Centre for Paediatric Pharmacy Research at the School of Pharmacy, University of London, says the treatment “would be an important, convenient and welcomed alternative to treat seizures”.

The oral midazolam has been shown in four clinical studies to be either comparable or superior in both its effectiveness and speed of onset of action to the current standard treatment.

Thierry Darcis, ViroPharma’s Vice President and General Manager in Europe, says the positive opinion is a “great step” for a product that “addresses significant unmet need for families and caregivers”.

He added that the company is working closely with healthcare professionals across Europe to bring this “much-needed emergency medication” to market as soon as possible.

PUMA is a new type of marketing authorisation for products that are developed specifically for children. These medicines benefit from 10 years of market protection.

ViroPharma’s Cinryze (C1 inhibitor [human]) has been granted a Centralised Marketing Authorisation for patients with hereditary angioedema (HAE) for the prevention and treatment of angioedema attacks.

Following a positive opinion from the CHMP back in March, the treatment becomes the first and only C1 inhibitor approved in Europe which prevents the attacks of this potentially fatal disease.

Professor Konrad Bork, Department of Dermatology of Johannes Gutenberg University in Mainz, Germany, says the approval of Cinryze provides “a very important new option” for patients.

The treatment will now be available through all EU Member States, as well as in Norway, Iceland and Liechtenstein.

HAE is a rare, potentially life-threatening genetic disorder affecting at least 10,000 individuals in Europe. Cinryze helps control HAE by increasing functional C1 inhibitor activity.

“We are thrilled to bring this important new therapy to European HAE patients seeking a more normal life through the prevention and treatment of their attacks,” said Vincent Milano, ViroPharma’s President and CEO.