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Pharma's Orphan Drug Activity, An Infographic

More than two-fifths of new drugs approved in the US last year were orphan drugs, and there are more than 4,500 drugs in active development for rare diseases. Effective orphan drug legislation and incentivization programs introduced gradually around the world since the 1980s mean rare diseases are no longer a rare sighting in the pharma industry pipeline, as this infographic illustrates.

Rare Diseases Market Intelligence Commercial

AZ’s ZS-9 Targets Hyperkalemia Sector As It Nears EU Market

A potential near-blockbuster drug, AstraZeneca’s Lokelma (ZS-9), has received a positive opinion from Europe’s CHMP and, if approved, will soon have to justify best-in-class plaudits and a hefty acquisition price.

Approvals Market Intelligence Research & Development

Patient Death Casts Cloud Over Roche Emicizumab Hemophilia Trial

The death of a patient during the HAVEN 1 study of Roche's investigational hemophilia product, emicizumab, may be nothing to panic about, but it is increasing concerns over its likely impact on the potential blockbuster drug's label.

Clinical Trials Drug Safety Research & Development

UCB Predicts Briviact Sales Of €450m by 2026; Plans New Partnerships

UCB says its early to mid-stage pipeline contains 10 promising breakthrough molecules, some of which it aims to partner with other pharma players as part of its open innovation strategy.

Companies Sales & Earnings Research and Development Strategies
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Merck's Write-Down Of Phase II Nuc Illustrates Current Reality In HCV

While a dwindling patient base and pricing pressures are depleting the market opportunity in hepatitis C, Merck's decision also may result from regulatory dialogue potentially delaying the start of a Phase III study.

Business Strategies Commercial Deals

Novartis Combo On Track To Be First Targeted BRAF NSCLC Therapy

The EU's CHMP has granted a positive opinion for Novartis' combo of MEK inhibitor Mekinist and BRAF inhibitor Tafinlar for BRAF-positive non-small cell lung cancer patients.

Approvals Commercial Companies

Latest From Commercial

Viking Flexes Its Muscles Ahead Of Partnering Talks

The mid-stage company is approaching Phase II data read outs for two lead assets. It is looking to partner with larger pharmas, but is also pursuing orphan indications that it could develop independently.

Business Strategies Rare Diseases

Rexgenero: Tackling Critical Limb Ischemia With Cell Therapy Approach

Emerging Company Profile: Rexgenero is developing a cell therapy to treat critical limb ischemia and is in the process of raising €25m to fund the Phase III program on top of €60m the company has raised to date. CEO Joe Dupere spoke with Scrip's Mike Ward at the Biotech Showcase last month.

Commercial Business Strategies

Deal Watch: Teva's Sol Barer Leads Buyout Of IO Start-up NexImmune

Instead of helping raise a Series A, the Teva chairman and others acquire the preclinical company, convinced of its synthetic nanotech promise in immuno-oncology. Vifor and ChemoCentryx expand their orphan and renal disease collaboration, while Gilead out-licenses an addiction drug candidate.

Commercial Deals

Celltrion's Truxima First Biosimilar MAB In Oncology To Win EU Approval

Celltrion's version of Roche’s MabThera (rituximab), Truxima, has become the first biosimilar anticancer to win marketing authorization in Europe.

Approvals Biosimilars

Bayer Bets On Oncology Pipeline, Vows To Increase 2017 R&D Budget

Bayer has launched an internal oncology R&D unit to speed up development of its pipeline cancer therapies and ensure the company is first to market with its late-stage treatment candidates, head of pharma Dieter Weinand told Scrip on the sidelines of the pharma's annual results conference.

Cancer Research and Development Strategies

Gilead, With Two PRVs In Hand, Holds Options For Accelerating Late-Stage Pipeline

Likely candidates for use of Gilead's priority review vouchers include GS-4997, a Phase III candidate for NASH, and bictegravir, its second-generation integrase inhibitor for HIV. The virology specialist previously used a PRV successfully to accelerate approval of Odefsey.

Business Strategies Research and Development Strategies

Policy & Regulation Explore this Topic

New EU Nod For J&J/Genmab's Darzalex Brings It Closer To Rivals

The EU's CHMP has given the go ahead to extend the EU licence for Janssen/Genmab's Darzalex to include its use earlier in the treatment paradigm, bringing into line with the US label and its rivals.

Europe Drug Review Policy & Regulation

New FDA Revlimid Approval Extends Celgene's Myeloma Empire

Celgene is finally able to market its mainstay multiple myeloma therapy Revlimid for maintenance use after stem cell transplant, after the FDA further expanded its approved indications. The EU is set to follow suit following a CHMP positive opinion last month.

Approvals Policy & Regulation

Busy In-Tray Awaits New EFPIA Boss Nathalie Moll

Nathalie Moll is moving on from leading the biotech industry body EuropaBio to be the next director general of EFPIA, the federation that represents the R&D-based pharmaceutical industry in Europe. With issues such as the forthcoming Brexit negotiations, the EU’s inquiry into medicines access and pricing, and the implementation of the new Clinical Trials Regulation on the agenda, Moll will have a lot of weighty dossiers to deal with.

Policy & Regulation Leadership

Rosalind Franklin Institute May Calm Industry’s Brexit Fears

The setting up of the Rosalind Franklin Institute to link up with one of the world’s largest electron microscopy centers in Harwell, Oxford, could bolster company sentiment towards UK R&D investment in a post-Brexit world.

Innovation Policy

Research & Development Explore this Topic

OncoSec Pushes IL-12 Approach Forward In PD-1 Melanoma Failures

Company plans to pursue accelerated approval based on a single-arm registrational study of PD-1 failures, after reporting positive results from a Phase II study at the ASCO-SITC meeting.

Clinical Trials ImmunoOncology Cancer

Latest From Research & Development

Ocular Therapeutix Gets Lead Product Back On Track

The long-acting eye insert Dextenza has been accepted for review by the US FDA, a piece of good news for Ocular Therapeutix that had previously received a complete response letter on manufacturing issues and a mixed bag of late-stage clinical trial results.

Companies Ophthalmic

Argos And Agenus Trials Pile On Fresh Cancer Vaccine Disappointments

Argos mum on details surrounding Phase III failure of rocapuldencel-T in kidney cancer, while Agenus distances itself from failed Phase II glioblastoma study of Prophage G-200 as an investigator-sponsored trial with a challenging design.

Research & Development Clinical Trials

What Progress In The Rare Disease, Osteogenesis Imperfecta?

Two-year-old biotech Mereo BioPharma Group plc is planning to start a pivotal Phase IIb study in osteogenesis imperfecta patients in the first-half of this year with its anti-sclerostin antibody, BPS-804, and if the results are promising, EU patients could gain early access through the region’s adaptive pathways program.

Rare Diseases Clinical Trials
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