EMA hands Italy's Chiesi a win in its long cam­paign to ad­vance new drugs for rare dis­eases

Five years af­ter Italy’s Chiesi built up its rare dis­ease ef­forts through ac­quir­ing Zymenex, the EMA has come through with a mar­ket­ing OK for a drug ob­tained in that deal.

Lamzede is an en­zyme re­place­ment ther­a­py for the ul­tra-rare al­pha-man­nosi­do­sis, a mul­ti-faceted de­bil­i­tat­ing dis­ease that is es­ti­mat­ed to af­fect 470 peo­ple in the EU. The treat­ment is ap­proved specif­i­cal­ly for non-neu­ro­log­i­cal man­i­fes­ta­tions in pa­tients with mild to mod­er­ate forms of the dis­or­der, a first for this pa­tient pop­u­la­tion.

The dis­ease is caused by a gene mu­ta­tion that leads to se­vere de­fi­cien­cy in al­pha-man­nosi­dase, an en­zyme that breaks down gly­co­pro­teins. As a re­sult, a tox­ic build-up of sug­ars takes place in the body, caus­ing a va­ri­ety of symp­toms from skele­tal ab­nor­mal­i­ties and mus­cle prob­lems to hear­ing loss and mo­tor skill im­pair­ments. Cur­rent treat­ments in­clude bone mar­row trans­plant and pe­riph­er­al blood stem cell trans­plan­ta­tion, in ad­di­tion to oth­er ther­a­pies tar­get­ing in­di­vid­ual symp­toms.

Alessan­dro Chiesi

Des­ig­nat­ed an or­phan drug back in 2005 when it was still in Zymenex’s hands, Chiesi’s Lamzede works by de­liv­er­ing, in­tra­venous­ly, a re­com­bi­nant form of al­pha-man­nosi­dase dubbed vel­manase al­fa to pa­tients. Falling un­der “ex­cep­tion­al cir­cum­stances,” the treat­ment’s ap­proval came af­ter sev­er­al clin­i­cal stud­ies in­volv­ing a to­tal of 34 pa­tients and a pos­i­tive CHMP opin­ion in Jan­u­ary.

The largest of those tri­als, a Phase III, en­rolled 25 pa­tients, and found re­duc­tion in serum oligosac­cha­rides for both the adult and pe­di­atric sub-groups. Those un­der 18 showed pos­i­tive im­prove­ment in func­tion­al tests like the three-minute stair climb­ing test and six-minute walk­ing test, while the adult group achieved sta­bi­liza­tion com­pared to the place­bo arm (who ex­pe­ri­enced more de­cline than the drug arm).

“Lamzede is the first ap­proved treat­ment with dis­ease mod­i­fy­ing po­ten­tial for al­pha-man­nosi­do­sis and there­fore, our next goal is to make this treat­ment avail­able to pa­tients in Eu­rope as soon as pos­si­ble,” said Alessan­dro Chiesi, head of Eu­rope at the Chiesi Group. “The mar­ket­ing au­tho­ri­sa­tion of Lamzede in the EU is a sig­nif­i­cant step­ping stone for pa­tients suf­fer­ing from this dev­as­tat­ing dis­ease and re­in­forces the Chiesi Group’s role in the com­plex and chal­leng­ing world of rare dis­eases.”

Chiesi build­ing in Ger­many. CHIESI DEUTSCH­LAND

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.