Pharmaceutical Technology Editors

Roche has announced in a March 30, 2021 press release, that the European Commission (EC) has approved its treatment for 5q spinal muscular atrophy (SMA), Evrysdi (risdiplam), in patients aged two months and older with a clinical diagnosis of SMA Type 1, Type 2, or Type 3, or with one to four SMD2 copies.

The approval decision was made based on data from two clinical studies in which Evrysdi demonstrated a favorable efficacy and safety profile in a broad spectrum of patients living with SMA. Roche is now working with health authorities across the European Union (EU) to achieve extensive and rapid access to the therapy for patients with SMA.

“Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU,” said Levi Garraway, Roche’s chief medical officer and head of Global Product Development, in the press release. “By avoiding the need for in-hospital administration, Evrysdi can reduce the treatment burden on those living with SMA, their caregivers, and healthcare systems. We thank the SMA community for their partnership, the trust they have placed in us and their unyielding commitment to achieve this significant milestone.”

“We welcome today’s approval of Evrysdi for people with SMA in Europe, and are proud of the role we have played in its development and of our partnership with Roche,” added Dr Nicole Gusset, president of SMA Europe, in the press release. “A recent survey conducted by SMA Europe showed that a large proportion of people with SMA in the EU were not receiving an approved treatment which leaves them feeling helpless and frustrated. It is vital that we work together with health authorities, regulators, and industry to ensure we can get this medicine to the patients who need it as soon as possible.”

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The EC has approved Roche's treatment for 5q spinal muscular atrophy (SMA), Evrysdi (risdiplam).

EC Approves Roche’s Home Treatment for SMA

Ofwat, a non-ministerial government department of the United Kingdom, and Nesta Challenges, part of the Nesta innovation foundation, have called on pharmaceutical innovators to collaborate with water companies for the new Water Breakthrough Challenge.

According to a March 25, 2021 press release, the £40 million (US $55 million) is being funded through Ofwat’s Innovation Fund, as part of the regulator’s goal to create an innovative and collaborative water sector that meets the needs of customers, society, and the environment. Through encouraging collaborations, the challenge aims to tackle the biggest obstacles facing water and wastewater services.

Entries are to be submitted by water companies in England and Wales, but partnerships with organizations outside the water sector, such as pharmaceuticals, are allowed. The open date for entries is May 6, 2021, and successful partnerships will have a chance to win up to £10 million (US $13.8 million).

“This competition follows on from the successful Innovation in Water Challenge that we ran earlier this year, when we received dozens of brilliant entries,” said John Russell, senior director at Ofwat, in the press release. “We now want to build on this momentum by encouraging and developing new partnerships and even bigger innovative projects that could make a real and lasting difference to the environment, society, and people’s lives—now and well into the future.”

“Climate change, extreme weather, and aging infrastructure are among the major challenges facing the water sector. Rising to these challenges demands that the sector goes beyond its business as usual, by seeking out, testing, and scaling up new solutions, new ways of working, and novel partnerships,” added Chris Gorst, director of Challenges at Nesta Challenges, in the press release. “With this Challenge we hope to pump-prime this process and to uncover and support truly breakthrough innovations that will deliver tangible benefits to water customers, wider society, and the environment.”

UK's Ofwat and Nesta Challenges have called on pharmaceutical innovators to collaborate with water companies for the new Water Breakthrough Challenge.

Innovators in Pharmaceuticals Are Being Called to Collaborate in New Water Challenge

The National Institute for Health and Care Excellence (NICE) has recommended carfilzomib in combination with lenalidomide and dexamethasone (KRd) as a treatment for adult patients with multiple myeloma who have already had one previous therapy, which included bortezomib.

This recommendation was based on positive clinical trial data, which demonstrated that the triple therapy provided longer periods of remission and allowed for longer life in patients when compared with the current second line treatment—lenalidomide plus dexamethasone. To make the combination therapy available, the drug manufacturer, Amgen, and the National Health Service have come to a confidential financial agreement.

“The recommendation of our committee will be welcomed by people with multiple myeloma who have told us of the need of a new second line treatment option that gives longer periods of remission and improves survival,” said Meindert Boysen, deputy CEO and director of the Centre for Health Technology Evaluation at NICE, in a March 22, 2021 press release. “The clinical data shows that the benefits of this triple therapy continue after treatment has stopped. A positive decision has been made possible after the company and NHS England came to a commercial arrangement which allows carfilzomib to be used on the NHS with a confidential discount.”

Tony Patrikios, executive medical director at Amgen UK and Ireland said, in a March 23, 2021 company release, “The relapsing-remitting nature of multiple myeloma can be psychologically very difficult for patients. Working collaboratively with our partners in the NHS and patient advocacy groups, there has been a strong tenacity to make this treatment combination cost effective and available to patients with multiple myeloma. We welcome this recommendation from NICE which provides another treatment option for patients whose disease has relapsed.”

NICE has recommended carfilzomib in combination with lenalidomide and dexamethasone (KRd) as a treatment for adult patients with multiple myeloma who have already had one previous therapy.

NICE Recommends Second Line Triple Therapy for Multiple Myeloma Patients

Ajinomoto Bio-Pharma Services (Aji Bio-Pharma), announced on March 30, 2021 that it has expanded its manufacturing agreement with AstraZeneca to include drug product manufacturing.

According to an Aji Bio-Pharma press release, the expanded agreement will include aseptic fill/finish services at its San Diego, CA facility, in addition to the small molecule manufacturing services already performed in Belgium.

"We are excited to expand our manufacturing agreement with AstraZeneca and to support them with additional service offerings," said Magnus Busch, global account manager, Ajinomoto Bio-Pharma Services, in the press release. "Our San Diego team is proud to join our Belgium colleagues in support of AstraZeneca. This expanded agreement builds on our commitment to be a leading, trusted, innovative partner to our clients."

The expanded agreement will include aseptic fill/finish services at its San Diego, CA facility.

Aji Bio-Pharma Expands Agreement with AstraZeneca to Include Drug Product Manufacturing

Novavax, GSK, and the UK Government Vaccines Taskforce announced on March 29, 2021 that they have reached an agreement in principle to support manufacturing of up to 60 million doses of Novavax' COVID-19 vaccine candidate (NVX-CoV2373) for use in the United Kingdom, purchased in advance by the UK government. GSK will provide fill/finish manufacturing capacity at its Barnard Castle facility in the northeast of England beginning as early as May 2021, and technology transfer will begin immediately. The specialized facility currently supports production of GSK pharmaceutical and vaccine products. According to the press release, GSK and Novavax will negotiate a final agreement to include additional terms and conditions.

The protein antigen component of NVX-CoV2373 is also produced in the northeast of England by Novavax' manufacturing partner, Fujifilm Diosynth Biotechnologies, at their site in Billingham, Stockton-on-Tees.

"GSK is delighted to support Novavax and the UK Vaccines Taskforce with this manufacturing arrangement for the UK, and our Barnard Castle facility is now undertaking the rapid preparation work required to manufacture up to 60 million doses of this vaccine,” said Roger Connor, president, GSK vaccines, in the press release. “We have ensured that we can deliver these volumes without impacting supply of our other vital medicines and vaccines, and without disruption to the other COVID-19 collaborations GSK is engaged in globally."

"This partnership with GSK continues the expansion of our global supply network, which we expect to increase overall production capacity and, if approved by regulatory agencies, support access to a potentially important new vaccine against COVID-19," said Rick Crowley, executive vice-president and chief operations officer, Novavax, in the press release.

GSK will provide filling capacity at its Barnard Castle facility in England.

GSK to Support Manufacture of Novavax' COVID-19 Vaccine Candidate

Biopharmaceutical company CureVac and contract development and manufacturing organization (CDMO) Celonic Group announced in a March 30, 2021 press release that they have entered into a commercial supply agreement to produce CureVac’s mRNA-based COVID-19 vaccine candidate, CvnCoV, at Celonic’s commercial manufacturing facility for biologics and advanced therapy medicinal products (ATMPs) in Heidelberg, Germany.

Celonic will be prepared to manufacture more than 100 million doses of CvnCoV, with more than 50 million doses expected to be produced before the end of 2021. Under the terms of the initial agreement, technology and knowledge transfer is already underway. The commercial supply agreement includes manufacturing of the mRNA drug substance as well as lipid nanoparticle formulation of the bulk drug product.

CureVac’s overall capacity, throughout its European manufacturing network, is expected to be up to 300 million doses in 2021.

“Manufacturing of sufficient quantities of vaccine is critical to combating the COVID-19 pandemic,” said Dr. Florian von der Mülbe, chief production officer of CureVac, in the press release. “With this partnership, we are further extending our integrated European manufacturing network, reinforcing the overall production capacity for our COVID-19 vaccine candidate, CVnCoV.”

“We have invested heavily to support our partners in bringing novel COVID-19 therapeutics and vaccines to patients, at an accelerated pace,” added Dr. Konstantin Matentzoglu, CEO of Celonic, in the press release.

Celonic will manufacture CvnCoV at its facility in Germany.