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.

Am­gen lays off about 300 work­ers, cit­ing 'in­dus­try head­wind­s'

Amgen has laid off about 300 employees, a company spokesperson confirmed to Endpoints News via email Sunday night.

Employees posted to LinkedIn in recent days about layoffs hitting Amgen last week. The Thousand Oaks, CA-based biopharma, which employs about 24,000 people, said the reduction “mainly” impacted US-based workers on its commercial team.

Drug developers of all sizes, including small upstarts and pharma giants, have let employees go in recent months as the biopharma market drags through a quarters-long winter doldrum.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bob Bradway, Amgen CEO (Stephen Lam/Reuters)

Am­gen launch­es the first US Hu­mi­ra biosim­i­lar at two dif­fer­ent list prices

The bizarre dynamics of the US prescription drug market were on full display once again this morning as Amgen announced that it would launch the first US biosimilar for Humira, the best-selling drug of all time, at two completely different list prices.

One price for Amgen’s Amjevita (adalimumab-atto) will be 55% below the current Humira list price, which is about $84,000 per year, and another at a list price 5% below the current Humira list price, but presumably (pharma companies don’t disclose rebates) with high rebates to attract PBMs and payers.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,800+ biopharma pros reading Endpoints daily — and it's free.

Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer Ingelheim uncorked some positive results suggesting that Spevigo can help prevent flare-ups in patients with a severe form of psoriasis, months after the drug was approved to treat existing flares.

Spevigo, an IL-36R antibody also known as spesolimab, met its primary and a key secondary endpoint in the Phase IIb EFFISAYIL 2 trial in patients with generalized pustular psoriasis (GPP), Boehringer announced on Monday. While the company is keeping the hard numbers under wraps until later this year, it said in a news release that it anticipates sharing the results with regulators.

As­traZeneca, No­vo Nordisk and Sanofi score 340B-re­lat­ed ap­peals court win over HHS

AstraZeneca, Novo Nordisk, and Sanofi won an appeals court win on Monday, as the US Court of Appeals for the Third Circuit found that the companies cannot be forced to provide 340B-discounted drugs purchased by hospitals from an unlimited number of community and specialty pharmacies.

“Legal duties do not spring from silence,” the decision says as the court makes clear that the federal government’s interpretation of the “supposed requirement” that the 340B program compels drugmakers to supply their discounted drugs to an unlimited number of contract pharmacies is not correct, noting:

Ap­peals court toss­es J&J's con­tro­ver­sial 'Texas two-step' bank­rupt­cy case

A US appeals court has ruled against Johnson & Johnson’s use of bankruptcy to deal with mounting talc lawsuits, deciding that doing so would “create a legal blind spot.”

The Third Circuit Court of Appeals reversed a previous bankruptcy court decision on Monday, calling for the dismissal of a Chapter 11 filing by J&J’s subsidiary LTL Management.

Faced with more than 38,000 lawsuits alleging its talc-based products caused cancer, J&J spun its talc liabilities into a separate company called LTL Management back in October 2021 and filed for bankruptcy, a controversial move colloquially referred to as a “Texas two-step” bankruptcy. Claimants argued that the strategy is a misuse of the US bankruptcy code — and on Monday, a panel of judges agreed.

Credit: Shutterstock

New York City in­vests $20M in­to biotech 'in­no­va­tion space' at the Brook­lyn Navy Yard

New York City is investing $20 million in biotech this year in the form of a 50,000-square-foot “innovation space” at the Brooklyn Navy Yard, complete with offices, research laboratories and events and programming space to grow biotech startups and companies.

Mayor Eric Adams said during his State of The City Address last Thursday that there will be an “emphasis” on making more opportunities for women and people of color to further diversify the industry. The City first reported the news.

Dirk Thye, Quince Therapeutics CEO

Af­ter piv­ot­ing from Alzheimer's to bone con­di­tions, biotech piv­ots again — and halves its head­count

When troubled public biotech Cortexyme bought a private startup named Novosteo and handed the keys to its executive team, the company — which changed its name to Quince Therapeutics — said it would shift its focus from an unorthodox Alzheimer’s approach to Novosteo’s bone-targeting drug platform.

Less than a year later, Quince is pivoting again.

The biotech has decided to out-license its bone-targeting drug platform and its lead drug, NOV004, and instead look for clinical-stage programs to in-license or acquire, according to a press release.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,800+ biopharma pros reading Endpoints daily — and it's free.

Troy Tazbaz, FDA's newly-named director of the Digital Health Center of Excellence (Oracle via YouTube)

Or­a­cle ex­ec­u­tive Troy Tazbaz named new FDA di­rec­tor of dig­i­tal health

The FDA has found a brand new director of the Digital Health Center of Excellence in Troy Tazbaz, a former senior vice president at Oracle.

According to Tazbaz’s LinkedIn, he took a five-month break after leaving an 11-year career at Oracle before joining the FDA in January. Stat News first reported the hire. Tazbaz also said on his LinkedIn that he biked all the way from Chesapeake Bay to the San Francisco Bay over 58 days during his career break.

Richard Gonzalez, AbbVie CEO (Chris Kleponis/picture-alliance/dpa/AP Images)

Up­dat­ed: $100B+ in sav­ings? Why the in­com­ing Hu­mi­ra biosim­i­lars will take time to catch on

The 20-year reign of AbbVie’s best-selling biologic of all time — the autoimmune disease biologic Humira (adalimumab) that has brought in upwards of $200 billion during its monopoly — is coming to an end tomorrow with the launch of Amgen’s biosimilar Amjevita.

The launch comes more than four years after Europe saw the exact same competition, leading to steep discounts in price, higher uptake, and big cost savings across the board.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,800+ biopharma pros reading Endpoints daily — and it's free.