EMA changes or­phan des­ig­na­tion pol­i­cy for cer­tain eye drugs to ex­pand ac­cess

The Eu­ro­pean Med­i­cines Agency said yes­ter­day that it’s chang­ing how it signs off on or­phan des­ig­na­tions for drugs tar­get­ing in­her­it­ed reti­nal dy­s­tro­phies (IRD) in or­der to make sure that pa­tients are not left out be­cause of the lim­i­ta­tions of the pre­vi­ous or­phan des­ig­na­tion terms.

In­her­it­ed reti­nal dy­s­tro­phies are a group of ge­net­ic and pro­gres­sive blind­ing dis­eases.

The Com­mit­tee for Or­phan Med­i­c­i­nal Prod­ucts for the EMA an­nounced the changed pol­i­cy, which the or­ga­ni­za­tion said was sup­port­ed by in­put from ex­perts and pa­tients of IRD, as well as a re­view of sci­en­tif­ic lit­er­a­ture and 64 ac­tive or­phan des­ig­na­tions in the spe­cif­ic eye dis­eases in the EU.

“Based on a thor­ough re­view, sup­port­ed by a con­sul­ta­tion of IRD clin­i­cal ex­perts and pa­tients, the COMP has adopt­ed a new ap­proach for des­ig­nat­ing con­di­tions in IRDs,” the state­ment reads.

There are now three op­tions for com­pa­nies want­i­ng to sub­mit an or­phan drug ap­pli­ca­tion in­to four dif­fer­ent groups: non-syn­dromic, syn­dromic, in­her­it­ed choroidal dy­s­tro­phies and hered­i­tary vit­re­o­retinopathies.

If a ther­a­py is “rel­a­tive­ly broad­ly ap­plic­a­ble” in IRD, terms for the ap­pli­ca­tion can be se­lect­ed from any of the four new groups. But if a drug that can be broad­ly used can tar­get more than one of the four groups, mul­ti­ple or­phan des­ig­na­tions could be nec­es­sary.

For gene ther­a­pies de­vel­oped for eye dis­eases, the con­di­tion in the or­phan drug des­ig­na­tion ap­pli­ca­tion has to be built from the term “in­her­it­ed reti­nal dy­s­tro­phy due to dys­func­tion in the tar­get-gene.”

If a drug doesn’t fit in­to any of the four groups, an “oc­ca­sion­al sin­gu­lar or­phan des­ig­na­tion” may still be nec­es­sary for a non-gene ther­a­py drug.

But why the change? The com­mit­tee de­cid­ed that IRDs are far too com­plex to con­tin­ue to use tra­di­tion­al terms that were de­vel­oped when the ge­net­ics be­hind the dis­eases weren’t un­der­stood and were sole­ly based on symp­toms.

“Us­ing clas­si­cal­ly de­rived IRD names for the or­phan con­di­tion may mean that some oth­er­wise treat­able pa­tients may be out of scope of an ap­proved treat­ment if these pa­tients show dif­fer­ent signs and symp­toms to the clas­si­cal group,” the ex­pla­na­tion from the com­mit­tee reads.

From now on, the com­mit­tee says that any com­pa­nies sub­mit­ting or­phan drug des­ig­na­tion ap­pli­ca­tions “spec­i­fy the or­phan con­di­tion ap­plied for and ful­ly jus­ti­fy the cho­sen ap­proach.” Any com­pa­nies with ex­ist­ing or­phan des­ig­na­tions should con­sid­er mak­ing any changes in line with the new groups be­fore sub­mit­ting a mar­ket­ing au­tho­riza­tion ap­pli­ca­tion.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.

Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”