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Scrip Asks… What Will Happen In Orphan Drug Pricing In The Next Five Years?

Feb 28 is Rare Disease Day. To mark the occasion, Scrip asked experts the question: Do you see orphan drug pricing coming under pressure over the next five years?

Rare Diseases Pricing Strategies Market Access

Shire's Wellhoefer On Genetic Disease R&D And Expanding Access To Medicines

Scrip spoke with Shire's Head of Genetic Diseases, Medical Affairs, Hartmann Wellhoefer about upcoming milestones in the company's R&D pipeline and improving access to medicines for rare diseases after the Baxalta merger.

Commercial Business Strategies Government Payers

Stockwatch: Alexion, United Therapeutics, Intercept And "Alternative Facts"

Mid-cap biotech companies Alexion and United Therapeutics both missed fourth-quarter sales expectations and although Intercept beat analysts' estimates with $13.4m in sales, does that justify a $3bn valuation?

Sales & Earnings Commercial BioPharmaceutical

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
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Stada Bows To Activist AOC, Opens Bidding Process To Three Suitors

Stada's supervisory board says it will play cards the way Active Ownership Capital wants for the time being, and has now opened the M&A game centered on the German group to a "structured bidding process".

Business Strategies M & A Companies

With Serial Entrepreneur At Its Helm, Arrakis Raises $38m To Target RNA With Small Molecules

Serial entrepreneur Michael Gilman has taken the helm of another start-up; after selling Padlock to Bristol-Myers and Stromedix to Biogen, his new company Arrakis has raised a $38m Series A round with big biopharma backing.

Commercial Companies Financing

Latest From Commercial

Astellas Builds Vaccine Portfolio With Affinivax Pneumococcal Candidate

Despite one recent setback, Astellas is continuing to expand its vaccines business through a new global deal for Affinivax's lead MAPS pneumococcal product, in a move that builds on earlier research links between the companies.

Vaccines Deals

Sanofi, Lonza JV Should Offer Pair Flexibility In Biologics Manufacturing

The pair's pact to build a biologics manufacturing site in Switzerland will allow Sanofi to expand or contract capacity usage in response to demand for its future biological therapies and give Lonza the option to pick up any eventual slack.

Biologics Business Strategies

Fed Up Of Poorly Run Academic Spin-Outs? Try This Instead

Three big pharma and three major UK universities are working together to "find, fund and run academic drug discovery projects at a pharma quality level." The collaboration has had a productive first year with four programs underway, two more about to be finalized, and "a good flow" in the pipeline, according to CEO Dr Richard Butt.

Research and Development Strategies Companies

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

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

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

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

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

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

Research & Development Explore this Topic

Merck & Co's New Shingles Vaccine Clears First Phase III Hurdle

Merck & Co has positive data from the first of two Phase III trials of its new varicella zoster virus vaccine for the prevention of shingles in immunocompromised patients, but will the product be enough to prop up its waning Zostavax in the face of GSK's Shingrix?

Clinical Trials Companies Vaccines

Latest From Research & Development

Pipeline Watch: Phase III Read-Outs For Ozanimod, Olaparib And Sapacitabine

Pipeline Watch has been updated to bring greater depth and detail to a potential therapeutic product’s progress through the R&D pipeline. The table brings you a snapshot of selected late-stage clinical trial events in the pharma and biotech industries.

Pipeline Watch Approvals

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

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
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